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    Clinical Trial Results:
    A Phase 2, Multicenter, Non-Randomized, Open-Label Study of RVT-1401 for the Treatment of Patients with Warm Autoimmune Hemolytic Anemia

    Summary
    EudraCT number
    		 2019-003924-19
    Trial protocol
    		 ES   GB   HU   PL   BG  
    Global end of trial date
    01 Apr 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Aug 2022
    First version publication date
    03 Aug 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    RVT-1401-2003
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04253236
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Immunovant Sciences GmbH
    Sponsor organisation address
    320 West, 37th Street, 6th Floor, New York, United States, 10018
    Public contact
    Central Study Contact, Immunovant Sciences GmbH, 1 800-797-0414, clinicaltrials@immunovant.com
    Scientific contact
    Central Study Contact, Immunovant Sciences GmbH, 1 800-797-0414, clinicaltrials@immunovant.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
    11 May 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Apr 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To examine the effect of RVT-1401 on proportion of responders (defined as Hb level ≥10g/dL with at least a ≥2 g/dL increase from baseline without rescue therapy or blood transfusions in the previous two weeks). To assess the safety and tolerability of RVT-1401 in participants with warm autoimmune hemolytic anemia.
    Protection of trial subjects
    The study was conducted in accordance with the principles of the Declaration of Helsinki (as amended in Edinburgh, Tokyo, Venice, Hong Kong, and South Africa), International Council for Harmonisation (ICH) guidelines, and all of the applicable basic principles of “Good Clinical Practice,” as outlined in 21 CFR 312, subpart D, “Responsibilities of Sponsors and Investigators,” 21 CFR, part 50, 1998, and 21 CFR, part 56, 1998. These standards were consistent with the requirements of the European Community Directive 2001/20/EC.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Aug 2020
    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
    United Kingdom: 1
    Country: Number of subjects enrolled
    Korea, Republic of: 3
    Country: Number of subjects enrolled
    Thailand: 1
    Worldwide total number of subjects
    5
    EEA total number of subjects
    0
    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)
    4
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 11 participants were screened, of which 5 participants were enrolled into the study. Due to the small number of participants (n=5) enrolled in the study, efficacy and safety conclusions could not be drawn and Pharmacokinetics/Pharmacodynamics (PK/PD) data were limited.

    Pre-assignment
    Screening details
    		 The study was terminated early prior to completion of dosing all participants in Cohort 1 and prior to initiating Cohort 2 due to a voluntary program-wide dosing pause to investigate unanticipated abnormalities in lipid levels observed in Thyroid Eye Disease patients enrolled in Study RVT-1401-2001 (NCT03938545).

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

    Arms
    Arm title
    		 Cohort 1: RVT-1401 680 mg/Week
    Arm description
    		 Participants received RVT-1401 680 milligram (mg) subcutaneous (SC) injection weekly for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    RVT-1401
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received 680 mg in two syringes of 2 milliliters (mL) RVT-1401 for a total of 4 mL.

    Number of subjects in period 1
    		Cohort 1: RVT-1401 680 mg/Week
    Started
    5
    Completed
    2
    Not completed
    3
         Adverse event, non-fatal
    1
         Safety Concerns
    1
         Study Terminated by Sponsor
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1: RVT-1401 680 mg/Week
    Reporting group description
    		 Participants received RVT-1401 680 milligram (mg) subcutaneous (SC) injection weekly for 12 weeks.

    Reporting group values
    		 Cohort 1: RVT-1401 680 mg/Week Total
    Number of subjects
    		 5 5
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 4 4
        From 65-84 years
    		 1 1
    Gender categorical
    Units: Subjects
        Female
    		 3 3
        Male
    		 2 2
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 4 4
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 1 1
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 5 5
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1: RVT-1401 680 mg/Week
    Reporting group description
    		 Participants received RVT-1401 680 milligram (mg) subcutaneous (SC) injection weekly for 12 weeks.

    Primary: Number of Responders at Week 13

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    End point title
    		 Number of Responders at Week 13 [1]
    End point description
    Responders were defined as the participants with level of hemoglobin (Hb) >=10 grams per deciliter (g/dL) with at least a >=2 g/dL increase from Baseline without rescue therapy or blood transfusions in the previous two weeks. Safety population: All participants who enrolled in the study and received at least 1 dose of study treatment. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Primary
    End point timeframe
    Week 13
    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: Statistical Analysis is not available.
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
    1
    No statistical analyses for this end point

    Primary: Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death

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    End point title
    		 Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death [2]
    End point description
    AEs were defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Clinically significant changes determined by the Investigator such as vital signs, Electrocardiograms (ECGs), and clinical laboratory values were also reported as AEs. TEAEs were defined as AEs that either started on or after the date of the first dose of study drug. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition. Safety Population.Data was not collected for Cohort 2.
    End point type
    Primary
    End point timeframe
    Up to Week 20
    Notes
    [2] - 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 performed for this safety endpoint.
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
        TEAEs
    5
        SAEs
    1
        Treatment-related AEs
    4
        Deaths
    0
    No statistical analyses for this end point

    Secondary: Time to Response

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    End point title
    		 Time to Response
    End point description
    The time to response was defined as the amount of time to achieve response (Hb levels >=10 g/dL with at least a >=2 g/dL increase from Baseline without rescue therapy or blood transfusions in the previous 2 weeks). Safety Population. Only those participants with data available at the specified time points were analyzed. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 13
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    1
    Units: Weeks
        number (not applicable)
    3
    No statistical analyses for this end point

    Secondary: Time to Achieving Hb Levels in the Normal Range

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    End point title
    		 Time to Achieving Hb Levels in the Normal Range
    End point description
    Time to achieving Hb levels in the normal range was assessed. Safety Population. Only those participants with data available at the specified time points were analyzed. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 13
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    1
    Units: Weeks
        number (not applicable)
    5
    No statistical analyses for this end point

    Secondary: Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score

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    End point title
    		 Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score
    End point description
    The FACIT-F scale was a validated scale which measured the physical, emotional and social implications of fatigue, one of the key clinical manifestations of warm autoimmune hemolytic anemia. Scores ranged from 0-52, a higher score indicated a higher quality of life. A score of less than 30 indicated severe fatigue. The scale took approximately 5-10 minutes to complete. Safety Population. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 13
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
    3
    No statistical analyses for this end point

    Secondary: Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale

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    End point title
    		 Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale
    End point description
    The MRC Breathlessness scale is a questionnaire that consisted of 5 statements about perceived Breathlessness and the focus of the scale was to quantify the disability associated with breathlessness. Score ranged from Grade 0 (limited to no disability) to Grade 4 (severe disability); higher score indicated severe disability. Safety Population. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 13
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
    4
    No statistical analyses for this end point

    Secondary: Number of Participants With Change in Euro Quality-5 dimension-3 level (EQ-5D-3L) Score

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    End point title
    		 Number of Participants With Change in Euro Quality-5 dimension-3 level (EQ-5D-3L) Score
    End point description
    The EQ-5D-3L is a validated measurement of health-related quality of life. The scale consists of 2 components, the EQ-5D descriptive system and the EQ visual analogue scale (VAS). The descriptive system evaluates mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems; a lower score indicated better quality of life. The EQ VAS records the participant’s self-rated health on a vertical visual analogue scale where the endpoints are labelled ‘Best imaginable health state’ (100) and ‘Worst imaginable health state’ (0). Safety Population. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 20
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
    4
    No statistical analyses for this end point

    Secondary: Concentration of RVT-1401 Pre-dose

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    End point title
    		 Concentration of RVT-1401 Pre-dose
    End point description
    Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic (PK) RVT-1401. Safety Population. Data could not be calculated due to high proportion of non-quantifiable values (>30% of values were imputed). Data was not collected for Cohort 2 due to early termination of the trial. 99999 indicates data is not available.
    End point type
    Secondary
    End point timeframe
    Pre-dose, Weeks 1, 2, 3, 4, 5, 6, 8, 10, 12 and 13 post-dose
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Milligrams per liter
        geometric mean (geometric coefficient of variation)
    99999 ( 99999 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Presence of Anti-RVT 1401 Antibodies

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    End point title
    		 Number of Participants With Presence of Anti-RVT 1401 Antibodies
    End point description
    Blood samples were collected at indicated time points to determine presence of anti-RVT 1401 antibodies. Participants with presence of anti-RVT 1401 antibodies is reported. Safety population. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Pre-dose on Weeks 1, 3, 5, 8, 13 and Week 20
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Change in Levels of Total Immunoglobulin (Ig)G and IgG Subclasses (1-4)

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    End point title
    		 Number of Participants With Change in Levels of Total Immunoglobulin (Ig)G and IgG Subclasses (1-4)
    End point description
    Blood samples were collected at indicated time points for pharmacodynamic (PD) analysis of serum total IgG and IgG subclasses (1-4) concentrations. Participants with changes in levels of Total IgG and IgG Subclasses (1-4) is reported. Safety Population. Data was not collected for Cohort 2 due to early termination of the trial.
    End point type
    Secondary
    End point timeframe
    Up to Week 20
    End point values
    		 Cohort 1: RVT-1401 680 mg/Week
    Number of subjects analysed
    5
    Units: Participants
        Total IgG
    5
        IgG Subclasses (1-4)
    5
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All-cause mortality, non-serious TEAEs and SAEs were collected up to Week 20 in Cohort 1.
    Adverse event reporting additional description
    Safety Population. Only data for Cohort 1 is presented as the study was terminated in Part 1; hence, Cohort 2 was not initiated.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Cohort 1: RVT-1401 680 mg/Week
    Reporting group description
    		 Participants received a RVT-1401 680 milligram (mg) subcutaneous (SC) injection weekly for 12 weeks.

    Serious adverse events
    		 Cohort 1: RVT-1401 680 mg/Week
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 5 (20.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Blood and lymphatic system disorders
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 5 (20.00%)
         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
    		 Cohort 1: RVT-1401 680 mg/Week
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Nervous system disorders
    Paraesthesia
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 5 (40.00%)
         occurrences all number
    3
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Oedema
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Oedema peripheral
         subjects affected / exposed
    2 / 5 (40.00%)
         occurrences all number
    2
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Gingival bleeding
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Hypoalbuminaemia
         subjects affected / exposed
    3 / 5 (60.00%)
         occurrences all number
    3
    Hypocalcaemia
         subjects affected / exposed
    1 / 5 (20.00%)
         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
    04 Oct 2019
    • Added anti-D, anti-Band 3, and/or anti-glycophorin antibodies to the Time and Events Table • Updated the pregnancy follow up language to create consistency with the safety management plan.
    23 Dec 2019
    • Updated WAIHA overview to provide clarity regarding worsening and refractory disease. • Updated Study Rationale to provide clarity regarding worsening and refractory disease. • Revised management criteria for hypoalbuminemia. • Added Exploratory Endpoint to evaluate transfusion burden • Updated to define therapy failure. • Revised to reflect management criteria for Grade 2-4 albumin levels. • Updated management criteria for infection • Updated management criteria for Grade 3 and 4 events to include study drug interruption and study drug discontinuation.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    02 Feb 2021
    In January 2021, Immunovant became aware of unanticipated abnormalities in lipid levels in Thyroid Eye Disease (TED) patients enrolled in Study RVT-1401-2001 (NCT03938545) during the conduct of the study and therefore, implemented a voluntary program-wide dosing pause to investigate the potential for batoclimab to affect lipids. Immunovant notified the FDA of the voluntary dosing pause in February 2021 and within this communication, committed to discussing a path forward with the Agency prior to resuming dosing in the batoclimab clinical development program. As a result of this voluntary pause, RVT-1401-2003 was paused, and an interim data cut occurred on 21 February 2021 to evaluate all efficacy and safety data and to inform the batoclimab program.
    -

    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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