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    Clinical Trial Results:
    A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria

    Summary
    EudraCT number
    		 2016-002652-25
    Trial protocol
    		 GB   IT  
    Global end of trial date
    14 Nov 2018

    Results information
    Results version number
    		 v3(current)
    This version publication date
    23 Jul 2022
    First version publication date
    23 May 2021
    Other versions
    		 v1 , v2
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    ACH471-100
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03053102
    WHO universal trial number (UTN)
    		 U1111-1190-3490
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    121 Seaport Boulevard, Boston, MA, United States, 02210
    Public contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 787-148-158, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 787-148-158, clinicaltrials.eu@alexion.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
    30 Oct 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    14 Nov 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Nov 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine the safety and efficacy of ACH-0144471 (danicopan) in currently untreated participants with paroxysmal nocturnal hemoglobinuria. After 12 weeks of treatment, participants deriving clinical benefit were offered enrollment in a separate long-term extension study (ACH471-103, 2017-000665-79).
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles outlined in the 2013 (Fortaleza, Brazil) revision of the 1964 Declaration of Helsinki, Good Clinical Practice (GCP) as required by the International Council of Harmonisation (ICH) guidelines, applicable regional and local laws legislation in the USA, and standard operating procedures (SOPs) in place.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    31 Mar 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 4 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    New Zealand: 6
    Country: Number of subjects enrolled
    Korea, Democratic People's Republic of: 1
    Worldwide total number of subjects
    10
    EEA total number of subjects
    2
    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)
    1
    Adults (18-64 years)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were evaluated for eligibility. A window of up to 60 days was permitted to allow screening followed by any required vaccinations.

    Pre-assignment
    Screening details
    		 In Part 1, participants received danicopan (100 or 150 mg TID) for 28 days. A dose increase up to 175 mg TID (N=8 participants) and 200 mg TID (N=4 participants) was conducted. Participants with reductions in lactate dehydrogenase (LDH) meeting specified criteria were offered continued dosing beyond Day 28, up to 8 additional weeks (Part 2).

    Period 1
    Period 1 title
    Study Part 1: 28-Day Treatment
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Danicopan
    Arm description
    		 Participants received danicopan 100 to 200 milligrams (mg) three times per day (TID).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    ACH-0144471
    Other name
    ACH-4471, ACH4471, 4471, ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    In Part 1, participants received danicopan for 28 days. Starting doses were 100 or 150 mg TID. A further dose increase up to 175 mg TID (N=8 participants) and 200 mg TID (N=4 participants) was conducted.

    Number of subjects in period 1
    		Danicopan
    Started
    10
    Completed
    10
    Period 2
    Period 2 title
    Study Part 2: 84-Day Treatment
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Danicopan
    Arm description
    		 Participants received danicopan 100 to 200 mg TID.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    ACH-0144471
    Other name
    ACH-4471, ACH4471, 4471, ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants with reductions in LDH meeting specified criteria in Part 1 were offered continued dosing beyond Day 28, for up to 8 additional weeks in Part 2. After 12 weeks of treatment, participants deriving clinical benefit were offered enrollment in a separate long-term extension study (ACH471-103, 2017-000665-79).

    Number of subjects in period 2
    		Danicopan
    Started
    10
    Completed
    8
    Not completed
    2
         Adverse event, serious fatal
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Study Part 1: 28-Day Treatment
    Reporting group description
    		 Participants received danicopan 100 to 200 mg TID.

    Reporting group values
    		 Study Part 1: 28-Day Treatment Total
    Number of subjects
    		 10 10
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 1 1
        Adults (18-64 years)
    		 9 9
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 35.94 ± 13.575 -
    Gender categorical
    Sex: Female, Male
    Units: Subjects
        Female
    		 5 5
        Male
    		 5 5
    Ethnicity
    Units: Subjects
        Not Hispanic or Latino
    		 10 10
        Hispanic or Latino
    		 0 0
        Unknown or Not Reported
    		 0 0
    Race
    Units: Subjects
        Asian
    		 1 1
        Native Hawaiian or Other Pacific Islander
    		 1 1
        White
    		 7 7
        Other
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    Danicopan
    Reporting group description
    		 Participants received danicopan 100 to 200 milligrams (mg) three times per day (TID).
    Reporting group title
    Danicopan
    Reporting group description
    		 Participants received danicopan 100 to 200 mg TID.

    Subject analysis set title
    Danicopan
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All participants who received at least 1 dose of study drug and had analysable data at the timepoints specified.

    Primary: Change From Baseline In Serum LDH Levels At Day 28

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    End point title
    		 Change From Baseline In Serum LDH Levels At Day 28 [1]
    End point description
    Change from Baseline = Serum LDH levels on Day 28 - Baseline Serum LDH levels.
    End point type
    Primary
    End point timeframe
    Baseline, Day 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: Only descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: international units per liter (IU/L)
    arithmetic mean (standard deviation)
        Baseline
    1416 ± 540.25
        Day 28 (Part 1)
    444.3 ± 255.78
        Change from Baseline
    -971.7 ± 549.76
    No statistical analyses for this end point

    Secondary: Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84

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    End point title
    		 Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84
    End point description
    Change from Baseline = Hgb levels on Days 28 or 84 - Baseline Hgb levels.
    End point type
    Secondary
    End point timeframe
    Baseline, Days 28 and 84
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: grams/deciliter (g/dL)
    arithmetic mean (standard deviation)
        Baseline
    9.76 ± 1.758
        Part 1: Day 28 (n=10)
    10.94 ± 1.651
        Part 1: Change from Baseline at Day 28 (n=10)
    1.18 ± 0.877
        Part 2: Day 84 (n=8)
    11.45 ± 1.414
        Part 2: Change from Baseline at Day 84 (n=8)
    1.80 ± 1.166
    No statistical analyses for this end point

    Secondary: Change From Baseline In Serum LDH Levels At Day 84

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    End point title
    		 Change From Baseline In Serum LDH Levels At Day 84
    End point description
    Change from Baseline = Serum LDH levels on Day 84 - Baseline Serum LDH levels. All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 84
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: IU/L
    arithmetic mean (standard deviation)
        Baseline (n=10)
    1416 ± 540.25
        Day 84 (Part 2) (n=8)
    537.3 ± 260.42
        Change from Baseline (n=8)
    -865.9 ± 447.86
    No statistical analyses for this end point

    Secondary: Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size

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    End point title
    		 Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size
    End point description
    PNH RBC, summed type III, clone size levels were assessed from Baseline to Day 28 and Day 84. The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 28, and Day 84
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: percentage of the total cell population
    arithmetic mean (standard deviation)
        Baseline (n=10)
    31.5363 ± 24.62004
        Day 28 (n=9)
    43.6279 ± 15.58021
        Day 84 (n=8)
    56.2953 ± 19.85086
    No statistical analyses for this end point

    Secondary: Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation

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    End point title
    		 Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation
    End point description
    An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Adverse Events section.
    End point type
    Secondary
    End point timeframe
    After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104)
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: participants
        SAE
    1
        TEAE Grade 3
    1
        TEAE Grade 4
    1
        AE leading to discontinuation
    1
    No statistical analyses for this end point

    Secondary: Grade 3 And Grade 4 Laboratory Abnormalities

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    End point title
    		 Grade 3 And Grade 4 Laboratory Abnormalities
    End point description
    Laboratory abnormalities were determined from laboratory measurements analyzed at the central or local laboratories, and were graded using CTCAE.
    End point type
    Secondary
    End point timeframe
    After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: participants
        Alanine aminotransferase increased
    1
        Aspartate aminotransferase increased
    1
        Low Hemoglobin
    1
        Low neutrophil count
    4
        High triglycerides
    1
    No statistical analyses for this end point

    Secondary: Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)

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    End point title
    		 Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)
    End point description
    Serial blood samples were collected predose and up to 8 hours postdose.
    End point type
    Secondary
    End point timeframe
    Days 6 and 20
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: hour (hr)*nanograms/milliliter (ng/mL)
    geometric mean (geometric coefficient of variation)
        Day 6: 100 mg TID (n=2)
    1432.611 ± 19.41
        Day 20: 150 mg TID (n=4)
    2370.421 ± 14.92
        Day 20: 175 mg TID (n=6)
    2278.038 ± 37.30
    No statistical analyses for this end point

    Secondary: PK: Maximum Plasma Concentration (Cmax)

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    End point title
    		 PK: Maximum Plasma Concentration (Cmax)
    End point description
    Serial blood samples were collected predose and up to 12 hours postdose.
    End point type
    Secondary
    End point timeframe
    Days 6 and 20
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Day 6: 100 mg TID (n=2)
    347.7 ± 9.57
        Day 20: 150 mg TID (n=4)
    583.6 ± 27.29
        Day 20: 175 mg TID (n=6)
    516.8 ± 33.39
    No statistical analyses for this end point

    Secondary: PK: Time To Maximum Concentration (Tmax)

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    End point title
    		 PK: Time To Maximum Concentration (Tmax)
    End point description
    Serial blood samples were collected predose and up to 12 hours postdose.
    End point type
    Secondary
    End point timeframe
    Days 6 and 20
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: hr
    median (full range (min-max))
        Day 6: 100 mg TID (n=2)
    4.17 (3.67 to 4.67)
        Day 20: 150 mg TID (n=4)
    3.67 (1.05 to 4.67)
        Day 20: 175 mg TID (n=6)
    4.11 (1.67 to 4.65)
    No statistical analyses for this end point

    Secondary: Complement Alternative Pathway (AP) Functional Activity

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    End point title
    		 Complement Alternative Pathway (AP) Functional Activity
    End point description
    Serum AP functional activity was measured by the Wieslab functional immunoassay method.
    End point type
    Secondary
    End point timeframe
    Baseline and Day 28
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: percentage of activity
    arithmetic mean (standard deviation)
        Baseline
    65.216 ± 13.7143
        Day 28
    12.730 ± 14.37670
    No statistical analyses for this end point

    Secondary: Complement Bb

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    End point title
    		 Complement Bb
    End point description
    Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA).
    End point type
    Secondary
    End point timeframe
    Baseline and Day 28
    End point values
    		 Danicopan
    Number of subjects analysed
    10
    Units: microgram (ug)/mL
    arithmetic mean (standard deviation)
        Baseline
    2.24160 ± 0.773962
        Day 28
    0.83913 ± 0.83821
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Danicopan
    Reporting group description
    		 Participants received danicopan 100 to 200 mg TID.

    Serious adverse events
    		 Danicopan
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 10 (10.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Danicopan
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 10 (90.00%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Vaccination site erythema
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Oedema peripheral
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Psychiatric disorders
    Irritability
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 10 (40.00%)
         occurrences all number
    5
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    3
    Paroxysmal nocturnal haemoglobinuria
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Gastrointestinal disorders
    Mouth ulceration
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Abdominal pain
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Rash papular
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Renal and urinary disorders
    Haemoglobinuria
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Myalgia
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Infections and infestations
    Pharyngitis
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 10 (40.00%)
         occurrences all number
    4
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Iron deficiency
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Mar 2017
    • Updated the dose levels based on the current PK modelling and clinical safety in healthy volunteers • Reworded the primary objective for clarity • Updated the previous human experience • Addressed discrepancies between this protocol and the ACH471-102 screening protocol
    19 Apr 2017
    • Upgraded the AE grading criteria used in the study to CTCAE and clarified language around the classification of AEs • Updated the previous human experience • Clarified the collection of information about red blood cell transfusions at each visit
    18 May 2017
    • Increased the starting dose from 100 mg TID (per the original protocol) to 150 mg TID • Increased the maximum permitted dose to 200 mg TID • Allowed vaccination concurrent with dosing, if local practice dictated
    05 Dec 2017
    • Updated the contraception section to include definitions requested by Health Authorities • Updated the contact information for SAE reporting • Added wording to permit the conducting of patient-reported outcomes interviews as questionnaires, where required
    21 Dec 2017
    • Increased the maximum permitted dose from 200 mg TID (Amendment 3) to 250 mg TID • Updated the contact information for SAE reporting
    13 Mar 2018
    • Specified that vaccination against bacterial infections should be performed, when necessary, based on vaccination history • Updated and clarified requirements for “acceptable” and “highly effecting” methods of contraception

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/33121236
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