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    Clinical Trial Results:
    A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

    Summary
    EudraCT number
    		 2020-005005-17
    Trial protocol
    		 HU  
    Global end of trial date
    18 Oct 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Nov 2024
    First version publication date
    02 Nov 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    R3918-PNH-2092
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04811716
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, NY, United States, 10591
    Public contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    18 Oct 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Oct 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP)
    Protection of trial subjects
    It is the responsibility of both the sponsor and the investigator(s) to ensure that this clinical study will be conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki, and that are consistent with the ICH guidelines for GCP and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Jul 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Hong Kong: 1
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Korea, Republic of: 12
    Country: Number of subjects enrolled
    Malaysia: 4
    Country: Number of subjects enrolled
    Taiwan: 4
    Country: Number of subjects enrolled
    United Kingdom: 2
    Worldwide total number of subjects
    24
    EEA total number of subjects
    1
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    19
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Twenty-four participants were screened and randomized.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pozelimab Q2W + Cemdisiran
    Arm description
    		 Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open¬ label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pozelimab
    Investigational medicinal product code
    REGN3918
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Administered by subcutaneous (SC) injection

    Arm title
    		 Pozelimab Q4W + Cemdisiran
    Arm description
    		 Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Cemdisiran
    Investigational medicinal product code
    ALN-CC5
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Administered by subcutaneous (SC) injection

    Number of subjects in period 1
    		Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Started
    12
    12
    Completed
    10
    12
    Not completed
    2
    0
         Consent withdrawn by subject
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pozelimab Q2W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open¬ label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.

    Reporting group title
    Pozelimab Q4W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.

    Reporting group values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran Total
    Number of subjects
    		 12 12 24
    Age Categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 41.4 ( 16.89 ) 53.2 ( 16.38 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 5 6 11
        Male
    		 7 6 13
    Race, Customized
    Units: Subjects
        White
    		 1 1 2
        Black or African American
    		 0 0 0
        Asian
    		 10 11 21
        American Indian or Alaska Native
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Not Reported
    		 1 0 1
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 12 12 24

    End points

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    End points reporting groups
    Reporting group title
    Pozelimab Q2W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open¬ label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.

    Reporting group title
    Pozelimab Q4W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.

    Subject analysis set title
    Pozelimab Q4W + Cemdisiran
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.

    Subject analysis set title
    Pozelimab Q4W + Cemdisiran
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.

    Primary: Percentage of participants with treatment emergent adverse events (TEAEs)

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    End point title
    		 Percentage of participants with treatment emergent adverse events (TEAEs) [1]
    End point description
    Safety analysis set (SAS) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'number analyzed' = the number of evaluable participants at a specified timepoint
    End point type
    Primary
    End point timeframe
    Through Week 28
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The analysis of the primary endpoint was descriptive i.e. no statistical hypothesis test was performed.
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
    number (not applicable)
        Participants with any TEAE
    66.7
    41.7
        Participants with serious TEAE
    16.7
    0
        Participants with severe TEAE
    16.7
    0
    No statistical analyses for this end point

    Secondary: Percent change of Lactate dehydrogenase (LDH) from pre-treatment to end-of-treatment period

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    End point title
    		 Percent change of Lactate dehydrogenase (LDH) from pre-treatment to end-of-treatment period
    End point description
    OLTP Pre-treatment (mean of LDH values prior to combination dosing); End-of-treatment (mean of LDH value at week 24- through week 28); Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); percentage of change in Upper Limit of Normal (xULN); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    End of treatment period, approximately 28 Weeks
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of change
        arithmetic mean (standard deviation)
    2.93 ( 36.575 )
    3.65 ( 13.608 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1) through Week 28
    End point description
    OLTP Adequate control of hemolysis is defined as LDH values ≤1.5 x Upper limit of normal (ULN) from baseline (day 1) to week 28; Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
        number (not applicable)
    75.0
    91.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants Maintaining Adequate Control of Hemolysis from Week 4 through Week 28

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    End point title
    		 Percentage of Participants Maintaining Adequate Control of Hemolysis from Week 4 through Week 28
    End point description
    OLTP Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Week 4 through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
        number (not applicable)
    83.3
    91.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Adequate Control of Hemolysis at Each Visit from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants with Adequate Control of Hemolysis at Each Visit from Baseline (Day 1) through Week 28
    End point description
    OLTP; Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
    number (not applicable)
        Baseline Visit (Day 1) n=12,12
    100.0
    100.0
        Pre-treatment n=12,12
    100.0
    100.0
        Week 1 n=12,12
    100.0
    100.0
        Week 2 n=12,12
    100.0
    100.0
        Week 4 n=12,12
    83.0
    100.0
        Week 6 n=12,12
    92.0
    100.0
        Week 8 n=12,11
    100.0
    100.0
        Week 10 n=11,12
    100.0
    100.0
        Week 12 n=12,12
    100.0
    92.0
        Week 16 n=12,12
    100.0
    100.0
        Week 20 n=12,12
    100.0
    100.0
        Week 24 n=12,12
    100.0
    100.0
        Week 28 n=12,12
    92.0
    100.0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Normalization of LDH at Each Visit from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants with Normalization of LDH at Each Visit from Baseline (Day 1) through Week 28
    End point description
    OLTP; Normalization of LDH was defined as LDH ≤1.0 × ULN at each visit; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
    number (not applicable)
        Baseline (Day 1) n=12,12
    92.0
    75.0
        Pre-treatment n=12,12
    92.0
    83.0
        Week 1 n=12,12
    100.0
    92.0
        Week 2 n=12,12
    92.0
    83.0
        Week 4 n=12,12
    83.0
    83.0
        Week 6 n=12,12
    83.0
    75.0
        Week 8 n=12,11
    100.0
    82.0
        Week 10 n=11,12
    100.0
    83.0
        Week 12 n=12,12
    92.0
    83.0
        Week 16 n=12,12
    92.0
    75.0
        Week 20 n=12,12
    100.0
    92.0
        Week 24 n=12,12
    100.0
    92.0
        Week 28 n=12,12
    83.0
    83.0
    No statistical analyses for this end point

    Secondary: Area under the curve (AUC) of LDH over time from Baseline (Day 1) through Week 28

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    End point title
    		 Area under the curve (AUC) of LDH over time from Baseline (Day 1) through Week 28
    End point description
    OLTP; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Units per Liter (U/L)
        arithmetic mean (standard deviation)
    238.18 ( 44.006 )
    244.42 ( 39.085 )
    No statistical analyses for this end point

    Secondary: AUC of LDH over time from Week 4 through Week 28

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    End point title
    		 AUC of LDH over time from Week 4 through Week 28
    End point description
    OLTP; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Week 4 through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: U/L
        arithmetic mean (standard deviation)
    202.49 ( 38.272 )
    211.28 ( 34.280 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Transfusion Avoidance from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants with Transfusion Avoidance from Baseline (Day 1) through Week 28
    End point description
    OLTP Not requiring a red blood cell (RBC) transfusion as per protocol algorithm; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
        number (not applicable)
    83.3
    100.0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1) through Week 28
    End point description
    OLTP Hemoglobin stabilization was defined as participants who did not receive an RBC transfusion and had no decrease in hemoglobin level of ≥2 grams per deciLiter (g/dL). FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
        number (not applicable)
    75.0
    91.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1) through Week 28

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    End point title
    		 Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1) through Week 28
    End point description
    OLTP; Breakthrough hemolysis = increase in LDH with concomitant signs or symptoms associated with hemolysis: • Increase in LDH occurs when: − LDH ≥2 × ULN if pre-treatment LDH is ≤1.5 × ULN or − LDH ≥2 × ULN after initial achievement of LDH ≤1.5 × ULN if pre-treatment LDH is >1.5 × ULN Signs/symptoms should correspond to those known to be associated with intravascular hemolysis due to Paroxysmal nocturnal hemoglobinuria (PNH) limited to the following: new onset or worsening fatigue, headache, dyspnea, hemoglobinuria, abdominal pain, scleral icterus, erectile dysfunction, chest pain, confusion, dysphagia, anemia including hemoglobin value significantly lower (ie, ≥2g/dL decrease) compared to participant’s baseline hemoglobin value, & thrombotic event. FAS = all randomized participants who received any study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Percentage of participants
        number (not applicable)
    8.3
    0.0
    No statistical analyses for this end point

    Secondary: Change in Hemoglobin Levels from Baseline (Day 1) through Week 28

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    End point title
    		 Change in Hemoglobin Levels from Baseline (Day 1) through Week 28
    End point description
    OLTP; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    11
    12
    Units: grams per Liter (g/L)
        arithmetic mean (standard deviation)
    -1.3 ( 13.77 )
    10.3 ( 7.41 )
    No statistical analyses for this end point

    Secondary: Change in Total complement hemolysis activity assay (CH50) from Baseline (Day 1) through Week 28

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    End point title
    		 Change in Total complement hemolysis activity assay (CH50) from Baseline (Day 1) through Week 28
    End point description
    OLTP; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Units per milliliter (U/mL)
        arithmetic mean (standard deviation)
    0.0 ( 0.0 )
    -0.1 ( 0.29 )
    No statistical analyses for this end point

    Secondary: Change in fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale from Baseline (Day 1) through Week 28

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    End point title
    		 Change in fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale from Baseline (Day 1) through Week 28
    End point description
    OLTP FACIT fatigue is a 13-item scale and for each item 4 is not at all fatigued to 0 very much fatigued. Higher FACIT-Fatigue scores indicate less fatigue (scores range from 0-52). A 5-point change is considered clinically meaningful; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    8
    9
    Units: Score on a scale
        arithmetic mean (standard deviation)
    -6.0 ( 10.09 )
    -2.09 ( 4.36 )
    No statistical analyses for this end point

    Secondary: Rate of Red Blood Cells (RBCs) transfused from Baseline (Day 1) to Week 28

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    End point title
    		 Rate of Red Blood Cells (RBCs) transfused from Baseline (Day 1) to Week 28
    End point description
    OLTP Rate of RBCs transfused is defined as number of events per patient-years. For each participant, the participant-years are the time from first dose date to week 28 (or early terminations visit if subject discontinued the study early) in the OLTP. FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Per participant-year of treatment
        number (confidence interval 95%)
    1.284 (0.169 to 9.760)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Number of Per-Protocol RBC Units Transfused from Baseline (Day 1) through Week 28

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    End point title
    		 Number of Per-Protocol RBC Units Transfused from Baseline (Day 1) through Week 28
    End point description
    OLTP; FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Units
        arithmetic mean (standard deviation)
    1.5 ( 3.73 )
    0.0 ( 0.00 )
    No statistical analyses for this end point

    Secondary: Change in global health status/quality of life scale (GHS/QoL) on the European Organization for Research and Treatment of Cancer: Quality-of-Life Questionnaire core 30 items (EORTC QLQ-C30) from Baseline (Day 1) through Week 28

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    End point title
    		 Change in global health status/quality of life scale (GHS/QoL) on the European Organization for Research and Treatment of Cancer: Quality-of-Life Questionnaire core 30 items (EORTC QLQ-C30) from Baseline (Day 1) through Week 28
    End point description
    OLTP; EORTC QLQ-C30 has 30 items (i.e. single questions), 24 of which are aggregated into 9 multi-item scales: 5 functioning scales (physical, role, cognitive, emotional & social), 3 symptom scales (fatigue, pain & nausea/vomiting) & 1 global health status scale. The remaining 6 single-item (dyspnoea, appetite loss, sleep disturbance, constipation, diarrhoea & the financial impact) scales assess symptoms. All of the scales & single-item measures range in score from 0-100. Higher score for functioning scales & global health status equal a better level of functioning (i.e. a better state of participant), while higher scores on the symptom & single-item scales indicate a higher level of symptoms (i.e. a worse state of the participant); FAS = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    8
    9
    Units: Score on a scale
        arithmetic mean (standard deviation)
    -9.4 ( 23.33 )
    -1.9 ( 13.03 )
    No statistical analyses for this end point

    Secondary: Concentrations of Total Pozelimab in Serum on Week 28

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    End point title
    		 Concentrations of Total Pozelimab in Serum on Week 28
    End point description
    OLTP; The PK analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.
    End point type
    Secondary
    End point timeframe
    On Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        arithmetic mean (standard deviation)
    131 ( 74.8 )
    58.2 ( 31.5 )
    No statistical analyses for this end point

    Secondary: Concentrations of Total C5 on Week 28

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    End point title
    		 Concentrations of Total C5 on Week 28
    End point description
    OLTP; The PK analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.
    End point type
    Secondary
    End point timeframe
    On Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        median (inter-quartile range (Q1-Q3))
    0 (0 to 13.8)
    0 (0 to 0)
    No statistical analyses for this end point

    Secondary: Concentrations of Cemdisiran in Plasma on Week 28

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    End point title
    		 Concentrations of Cemdisiran in Plasma on Week 28
    End point description
    OLTP; The PK analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.
    End point type
    Secondary
    End point timeframe
    On Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Change in physical function (PF) scores on the EORTC QLQ-C30 from Baseline (Day 1) through Week 28

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    End point title
    		 Change in physical function (PF) scores on the EORTC QLQ-C30 from Baseline (Day 1) through Week 28
    End point description
    OLTP; The EORTC QLQ-C30 is a 30-item, self-administered, generic questionnaire that assesses health-related QoL across multiple domains, including GHS, global QoL, functioning (physical, role, emotional, cognitive, and social functioning), symptom scales (fatigue, nausea and vomiting, pain, appetite loss), and single items (dyspnea, insomnia, constipation, diarrhea, sleep, financial impact). EORTC QLQ-C30 domain scales range from 0 to 100, with lower scores indicating worse QoL and higher scores for symptom scales indicating worse symptoms. A 10-point change is considered meaningful; Full analysis set (FAS) = included all randomized participants who eceived any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    8
    9
    Units: Score on a scale
        arithmetic mean (standard deviation)
    -6.7 ( 19.19 )
    0.7 ( 9.09 )
    No statistical analyses for this end point

    Secondary: Number of Participants with Cemdisiran Anti-Drug Antibody (ADA) Responses Over Time

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    End point title
    		 Number of Participants with Cemdisiran Anti-Drug Antibody (ADA) Responses Over Time
    End point description
    OLTP and OLEP; Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint
    End point type
    Secondary
    End point timeframe
    Up to Week 52
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Participants
    number (not applicable)
        Treatment-Boosted Response
    0
    0
        Treatment-Emergent Response
    0
    1
    No statistical analyses for this end point

    Secondary: Number of Participants with Pozelimab Anti-Drug Antibody (ADA) Responses Over Time

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    End point title
    		 Number of Participants with Pozelimab Anti-Drug Antibody (ADA) Responses Over Time
    End point description
    OLTP and OLEP; Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint
    End point type
    Secondary
    End point timeframe
    Up to Week 52
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: Participants
    number (not applicable)
        Treatment-Boosted Response
    0
    0
        Treatment-Emergent Response
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of participants with TEAEs for participants who received treatment intensification

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    End point title
    		 Percentage of participants with TEAEs for participants who received treatment intensification
    End point description
    OLTP No participants received dose intensification during the study; Therefore, assessment of the safety of pozelimab + cemdisiran combination therapy in participants requiring dose intensification was not conducted; Safety analysis set (SAS) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'n' = the number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Through Week 28
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: Percentage of participants
    number (not applicable)
        Participants with any TEAE
        Participants with any serious TEAE
        Participants with any severe TEAE
    Notes
    [2] - No participants received dose intensification; assessment was not conducted
    [3] - No participants received dose intensification; assessment was not conducted
    No statistical analyses for this end point

    Secondary: Change of LDH from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change of LDH from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: U/L
        arithmetic mean (standard deviation)
    -14.5 ( 105.90 )
    No statistical analyses for this end point

    Secondary: Percent Change of LDH from OLEP Baseline (Day 1e) to Week 24e

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    End point title
    		 Percent Change of LDH from OLEP Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: Percentage of change
        arithmetic mean (standard deviation)
    -0.7 ( 20.60 )
    No statistical analyses for this end point

    Secondary: Change of LDH from Baseline (Day 1e) to Week 24e

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    End point title
    		 Change of LDH from Baseline (Day 1e) to Week 24e
    End point description
    Optional Open-Label Extension Period (OLEP); OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: U/L
        arithmetic mean (standard deviation)
    -18.2 ( 108.40 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1e) through Week 24e

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    End point title
    		 Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1e) through Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of Participants
        number (not applicable)
    95.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants Maintaining Adequate Control of Hemolysis from Baseline (Day 1e) through Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of Participants
        number (not applicable)
    95.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Adequate Control of Hemolysis at Each Visit from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants with Adequate Control of Hemolysis at Each Visit from Baseline (Day 1e) through Week 52e
    End point description
    OLEP; Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
    number (not applicable)
        OLEP Baseline (Day 1e)
    96.0
        Week 8e
    100.0
        Week 16e
    100.0
        Week 24e
    100.0
        Week 32e
    100.0
        Week 40e
    100.0
        Week 52e
    100.0
    No statistical analyses for this end point

    Secondary: Percent change of LDH from Baseline (Day 1e) to Week 52e

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    End point title
    		 Percent change of LDH from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Percentage of change for units per liter (U/L); Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: Percentage of change
        arithmetic mean (standard deviation)
    0.2 ( 19.49 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Normalization of LDH at Each Visit from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants with Normalization of LDH at Each Visit from Baseline (Day 1e) through Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
    number (not applicable)
        OLEP Baseline (Day 1e)
    87.0
        Week 8e
    83.0
        Week 16e
    82.0
        Week 24e
    82.0
        Week 32e
    86.0
        Week 40e
    86.0
        Week 52e
    82.0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1e) through Week 24e

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    End point title
    		 Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1e) through Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of Participants
        number (not applicable)
    4.3
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants with Breakthrough hemolysis from Baseline (Day 1e) through Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
        number (not applicable)
    4.3
    No statistical analyses for this end point

    Secondary: AUC of LDH over time from Baseline (Day 1e) through Week 52e

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    End point title
    		 AUC of LDH over time from Baseline (Day 1e) through Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: U/L
        arithmetic mean (standard deviation)
    154.63 ( 38.231 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1e) through Week 24e

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    End point title
    		 Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1e) through Week 24e
    End point description
    OLEP Participants who do not receive RBC transfusion and have no decrease in hemoglobin levels; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
        number (not applicable)
    78.3
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants with Hemoglobin Stabilization from Baseline (Day 1e) through Week 52e
    End point description
    OLEP Participants who do not receive RBC transfusion and have no decrease in hemoglobin levels; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
        number (not applicable)
    69.6
    No statistical analyses for this end point

    Secondary: Change in hemoglobin levels from Baseline (Day 1e) to Week 24e

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    End point title
    		 Change in hemoglobin levels from Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    21
    Units: g/L
        arithmetic mean (standard deviation)
    0.8 ( 15.63 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Per-Protocol Transfusion Avoidance from Baseline (Day 1e) through Week 52e

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    End point title
    		 Percentage of Participants with Per-Protocol Transfusion Avoidance from Baseline (Day 1e) through Week 52e
    End point description
    OLEP Not requiring a RBC transfusion as per protocol algorithm; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of Participants
        number (not applicable)
    87.0
    No statistical analyses for this end point

    Secondary: Change in hemoglobin levels from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change in hemoglobin levels from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: g/L
        arithmetic mean (standard deviation)
    -0.6 ( 12.60 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Per-Protocol Transfusion Avoidance from Baseline (Day 1e) through Week 24e

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    End point title
    		 Percentage of Participants with Per-Protocol Transfusion Avoidance from Baseline (Day 1e) through Week 24e
    End point description
    OLEP Not requiring a RBC transfusion as per protocol algorithm; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) through Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of Participants
        number (not applicable)
    87.0
    No statistical analyses for this end point

    Secondary: Number of units of RBCs transfused from Baseline (Day 1e) to Week 24e

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    End point title
    		 Number of units of RBCs transfused from Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Units
        arithmetic mean (standard deviation)
    0.4 ( 1.47 )
    No statistical analyses for this end point

    Secondary: Rate of RBCs transfused from Baseline (Day 1e) to Week 52e

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    End point title
    		 Rate of RBCs transfused from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Per participant-year of treatment
        number (confidence interval 95%)
    0.506 (0.092 to 2.773)
    No statistical analyses for this end point

    Secondary: Change in CH50 from Baseline (Day 1e) to Week 16e

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    End point title
    		 Change in CH50 from Baseline (Day 1e) to Week 16e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 16e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: U/mL
        arithmetic mean (standard deviation)
    0.0 ( 0.00 )
    No statistical analyses for this end point

    Secondary: Rate of RBCs transfused from Baseline (Day 1e) to Week 24e

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    End point title
    		 Rate of RBCs transfused from Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Per participant-year of treatment
        number (confidence interval 95%)
    0.591 (0.143 to 2.445)
    No statistical analyses for this end point

    Secondary: Change in CH50 from Baseline (Day 1e) to Week 24e

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    End point title
    		 Change in CH50 from Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants have 0 values at baseline for CH50, the percentage change is not appropriate and undefined. Therefore, this endpoint is not able to be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    0 [4]
    Units: U/mL
        arithmetic mean (standard deviation)
    ( )
    Notes
    [4] - All participants have 0 values at baseline, percentage change is undefined and cannot be calculated.
    No statistical analyses for this end point

    Secondary: Number of units of RBCs transfused from Baseline (Day 1e) to Week 52e

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    End point title
    		 Number of units of RBCs transfused from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Units
        arithmetic mean (standard deviation)
    0.9 ( 3.95 )
    No statistical analyses for this end point

    Secondary: Change in GHS/QoL on the EORTC QLQ-C30 from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change in GHS/QoL on the EORTC QLQ-C30 from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; The EORTC QLQ-C30 is a 30-item, self-administered, generic questionnaire that assesses health-related QoL across multiple domains, including GHS, global QoL, functioning (physical, role, emotional, cognitive, and social functioning), symptom scales (fatigue, nausea and vomiting, pain, appetite loss), and single items (dyspnea, insomnia, constipation, diarrhea, sleep, financial impact). EORTC QLQ-C30 domain scales range from 0 to 100, with lower scores indicating worse QoL and higher scores for symptom scales indicating worse symptoms. A 10-point change is considered meaningful. OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    21
    Units: Score on a scale
        arithmetic mean (standard deviation)
    6.0 ( 13.73 )
    No statistical analyses for this end point

    Secondary: Change in fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change in fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; The FACIT-Fatigue is a 13-item, self-administered assessment of an individual’s level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related QoL in participants with cancer and other chronic illnesses. The FACIT-Fatigue items are measured with a 5-point Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating less fatigue. A 5-point change is considered clinically meaningful. OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    21
    Units: Score on a scale
        arithmetic mean (standard deviation)
    -0.9 ( 5.95 )
    No statistical analyses for this end point

    Secondary: Change in CH50 from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change in CH50 from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    22
    Units: U/mL
        arithmetic mean (standard deviation)
    0.0 ( 0.21 )
    No statistical analyses for this end point

    Secondary: Percent change in CH50 from Baseline (Day 1e) to Week 16e

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    End point title
    		 Percent change in CH50 from Baseline (Day 1e) to Week 16e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants have 0 values at baseline for CH50, the percentage change is not appropriate and undefined. Therefore, this endpoint is not able to be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 16e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    0 [5]
    Units: Percentage of change
        number (not applicable)
    Notes
    [5] - All participants have 0 values at baseline, percentage change is undefined and cannot be calculated.
    No statistical analyses for this end point

    Secondary: Percent change in CH50 from Baseline (Day 1e) to Week 52e

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    End point title
    		 Percent change in CH50 from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants have 0 values at baseline for CH50, the percentage change is not appropriate and undefined. Therefore, this endpoint is not able to be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    0 [6]
    Units: Percentage of change
        number (not applicable)
    Notes
    [6] - All participants have 0 values at baseline, percentage change is undefined and cannot be calculated.
    No statistical analyses for this end point

    Secondary: Percent change in CH50 from Baseline (Day 1e) to Week 24e

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    End point title
    		 Percent change in CH50 from Baseline (Day 1e) to Week 24e
    End point description
    OLEP; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants have 0 values at baseline for CH50, the percentage change is not appropriate and undefined. Therefore, this endpoint is not able to be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 24e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    0 [7]
    Units: Percentage of change
        number (not applicable)
    Notes
    [7] - All participants have 0 values at baseline, percentage change is undefined and cannot be calculated.
    No statistical analyses for this end point

    Secondary: Concentrations of Total Pozelimab in Serum on Week 52

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    End point title
    		 Concentrations of Total Pozelimab in Serum on Week 52
    End point description
    OLEP; The OLEP PK analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.
    End point type
    Secondary
    End point timeframe
    On Week 52
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        arithmetic mean (standard deviation)
    63.4 ( 35.6 )
    58.6 ( 28.7 )
    No statistical analyses for this end point

    Secondary: Change in PF scores on the EORTC QLQ-C30 from Baseline (Day 1e) to Week 52e

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    End point title
    		 Change in PF scores on the EORTC QLQ-C30 from Baseline (Day 1e) to Week 52e
    End point description
    OLEP; The EORTC QLQ-C30 is a 30-item, self-administered, generic questionnaire that assesses health-related QoL across multiple domains, including GHS, global QoL, functioning (physical, role, emotional, cognitive, and social functioning), symptom scales (fatigue, nausea and vomiting, pain, appetite loss), and single items (dyspnea, insomnia, constipation, diarrhea, sleep, financial impact). EORTC QLQ-C30 domain scales range from 0 to 100, with lower scores indicating worse QoL and higher scores for symptom scales indicating worse symptoms. A 10-point change is considered meaningful.; OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1e) to Week 52e
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    21
    Units: Score on a scale
        arithmetic mean (standard deviation)
    -0.3 ( 3.93 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with TEAEs Up to Week 52

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    End point title
    		 Percentage of Participants with TEAEs Up to Week 52
    End point description
    OLEP; SAS; Here 'n' = number of evaluable participants at a specified timepoint
    End point type
    Secondary
    End point timeframe
    Up to Week 52
    End point values
    		 Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    23
    Units: Percentage of participants
    number (not applicable)
        Participants with any TEAE
    73.9
        Participants with serious TEAE
    8.7
        Participants with severe TEAE
    4.3
    No statistical analyses for this end point

    Secondary: Concentrations of Total C5 on Week 52

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    End point title
    		 Concentrations of Total C5 on Week 52
    End point description
    OLEP; The OLEP PK analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.
    End point type
    Secondary
    End point timeframe
    On Week 52
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        median (inter-quartile range (Q1-Q3))
    0 (0 to 8.05)
    0 (0 to 0)
    No statistical analyses for this end point

    Secondary: Concentrations of Cemdisiran in Plasma on Week 52

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    End point title
    		 Concentrations of Cemdisiran in Plasma on Week 52
    End point description
    OLEP; The OLEP PK analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.
    End point type
    Secondary
    End point timeframe
    On Week 52
    End point values
    		 Pozelimab Q2W + Cemdisiran Pozelimab Q4W + Cemdisiran
    Number of subjects analysed
    12
    12
    Units: mg/L
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    1. OLTP (from first dose up to Week 28 + follow-up of participant without OLE, total 24 participants) 2. OLEP (from Week 28 EOT up to Week 52 OLEP and up to Week 52 follow-up EOS, total 23 participants)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    OLTP_Pozelimab Q2W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open¬ label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.

    Reporting group title
    OLEP_Pozelimab Q4W + Cemdisiran
    Reporting group description
    		 In the open-label extension period (OLEP), participants received a regimen of pozelimab SC Q4W + cemdisiran SC, regardless of their treatment assignment in the OLTP.

    Reporting group title
    OLTP_Pozelimab Q4W + Cemdisiran
    Reporting group description
    		 Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.

    Serious adverse events
    		 OLTP_Pozelimab Q2W + Cemdisiran OLEP_Pozelimab Q4W + Cemdisiran OLTP_Pozelimab Q4W + Cemdisiran
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 12 (25.00%)
    2 / 23 (8.70%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 23 (4.35%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Anal fistula
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 23 (4.35%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Large intestine polyp
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 23 (4.35%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 OLTP_Pozelimab Q2W + Cemdisiran OLEP_Pozelimab Q4W + Cemdisiran OLTP_Pozelimab Q4W + Cemdisiran
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 12 (66.67%)
    10 / 23 (43.48%)
    5 / 12 (41.67%)
    Investigations
    Free haemoglobin present
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Electrocardiogram ST segment depression
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Transaminases increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Injection site reaction
         subjects affected / exposed
    2 / 12 (16.67%)
    3 / 23 (13.04%)
    2 / 12 (16.67%)
         occurrences all number
    6
    4
    4
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Breakthrough haemolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    Ear and labyrinth disorders
    Inner ear disorder
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Anal fistula
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Renal and urinary disorders
    Paroxysmal nocturnal haemoglobinuria
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Arthralgia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Myalgia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    Muscle spasms
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    2 / 12 (16.67%)
    4 / 23 (17.39%)
    1 / 12 (8.33%)
         occurrences all number
    2
    4
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 12 (16.67%)
    4 / 23 (17.39%)
    0 / 12 (0.00%)
         occurrences all number
    2
    5
    0
    Subcutaneous abscess
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Chlamydial infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 23 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Dec 2021
    The main purpose of this amendment was to modify the timing of the interim analysis and to align endpoints with other studies.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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