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    Summary
    EudraCT Number:2020-002031-29
    Sponsor's Protocol Code Number:R7257-RAA-1947
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-06-30
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2020-002031-29
    A.3Full title of the trial
    A PHASE 1/2 STUDY OF REGN7257 (ANTI-INTERLEUKIN 2 RECEPTOR SUBUNIT GAMMA [IL2RG] MONOCLONAL ANTIBODY) IN PATIENTS WITH SEVERE APLASTIC ANEMIA THAT IS REFRACTORY TO OR RELAPSED ON IMMUNOSUPPRESSIVE THERAPY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    REGN7257 in Adult Patients with Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
    A.4.1Sponsor's protocol code numberR7257-RAA-1947
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04409080
    A.5.4Other Identifiers
    Name:INDNumber:147248
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRegeneron Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportRegeneron Pharmaceuticals, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.6E-mailClinicaltrials@regeneron.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code REGN7257
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Applicable
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeREGN7257
    D.3.9.3Other descriptive nameHUMAN IGG4 ISOTYPE MONOCLONAL (IL2-RG) ANTIBODY
    D.3.9.4EV Substance CodeSUB130944
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number265
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    SEVERE APLASTIC ANEMIA
    E.1.1.1Medical condition in easily understood language
    SEVERE APLASTIC ANEMIA
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002274
    E.1.2Term Anemia aplastic
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia (SAA) that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in IST-refractory/ relapsed patients.
    E.2.2Secondary objectives of the trial
    - Clinical response over time
    - Maintenance of response
    - Impact on transfusion requirements
    - Effect on blood counts and cell populations
    - Pharmacokinetics (PK)
    - Immunogenicity
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    1: Future Biomedical Research sub-study:
    Patients who agree to participate in the future biomedical research sub-study will be required to
    consent to this optional sub-study before samples are banked in long-term storage. Additional
    samples will be collected for future biomedical research according to time points listed in the
    Schedule of Events (Table 1 and Table 2). The unused biomarker samples for study-related
    research, the unused PK and ADA samples, as well as the samples collected for future biomedical
    research, will be stored for up to 15 years after the final date of the database lock. The unused
    samples may be utilized for future biomedical research.

    2: Pharmacogenomic Analysis sub-study:
    The purpose of the pharmacogenomic analyses is to identify genomic associations with clinical or
    biomarker response to REGN7257, other aplastic anemia clinical outcome measures and possible
    AEs.
    E.3Principal inclusion criteria
    1. SAA that is refractory to or has relapsed while on standard of care IST, as defined in the protocol
    2. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
    3. Adequate hepatic and renal function as defined in the protocol

    Other protocol-defined inclusion criteria apply
    E.4Principal exclusion criteria
    1. Diagnosis of Fanconi anemia as defined in the protocol
    2. Evidence of myelodysplastic syndrome as defined in the protocol
    3. Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis or history of PNH-associated thrombosis
    4. Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
    5. Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing
    6. Treatment with eltrombopag or investigational thrombopoietin receptor agonist, Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2 weeks prior to dosing
    7. HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit
    8. Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI

    Other protocol-defined exclusion criteria apply
    E.5 End points
    E.5.1Primary end point(s)
    Part A
    - Incidence of adverse events (AEs); 12 months post-treatment, approximately 52 weeks

    Parts A and B
    - Incidence of serious adverse events (SAEs); 12 months post-treatment, approximately 52 weeks
    - Incidence and severity of treatment-emergent adverse events (TEAEs); 12 months post-treatment, approximately 52 weeks

    Part B
    - Overall response rate (ORR); 6 months post-treatment, approximately 26 weeks
    E.5.1.1Timepoint(s) of evaluation of this end point
    See above
    E.5.2Secondary end point(s)
    Parts A and B
    ORR; 3 months post-treatment, approximately 12 weeks
    Complete response (CR); 3 months post-treatment, approximately 12 weeks
    Partial response (PR); 3 months post-treatment, approximately 12 weeks
    Time to best response; Up to 12 months
    Time to first response; Up to 12 months
    Proportion of patients who maintain any clinical response; Up to 12 months
    Platelet transfusions per month over time; Up to 12 months
    Red blood cell transfusions per month over time; Up to 12 months
    Changes in lymphocyte cell counts; Up to 12 months
    Changes in neutrophil cell counts; Up to 12 months
    Changes in hemoglobin cell counts; Up to 12 months
    Changes in reticulocyte cell counts; Up to 12 months
    Changes in platelet cell counts; Up to 12 months
    Changes in the whole blood immune cell subsets (T cells); Up to 12 months
    Changes in the whole blood immune cell subsets (B cells); Up to 12 months
    Changes in the whole blood immune cell subsets (NK cells); Up to 12 months
    Drug concentrations in serum over time; Up to 12 months
    Incidence of treatment-emergent anti-drug antibody (ADA) over time; Up to 12 months
    E.5.2.1Timepoint(s) of evaluation of this end point
    See above
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity and Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    Korea, Republic of
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 48
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-07-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-05
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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