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REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04409080
Recruitment Status : Recruiting
First Posted : June 1, 2020
Last Update Posted : June 27, 2022
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Study Description
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Brief Summary:

The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with immunosuppressive therapy (IST)-refractory or IST-relapsed severe aplastic anemia (SAA). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in patients with IST-refractory or IST-relapsed SAA.

The secondary objectives of this study are to assess the following for REGN7257:

  • Clinical response over time
  • Maintenance of response
  • Impact on transfusion requirements
  • Effect on blood counts and cell populations
  • Pharmacokinetics (PK)
  • Immunogenicity

Condition or disease Intervention/treatment Phase
Severe Aplastic Anemia (SAA) Drug: REGN7257 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 57 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : January 13, 2021
Estimated Primary Completion Date : August 8, 2024
Estimated Study Completion Date : August 8, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Part A and Part B
Part A: Single ascending dose Part B: Preferred dose
 Drug: REGN7257
Single dose will be administered by intravenous IV infusion


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of adverse events (AEs) [ Time Frame: 12 months post-treatment, approximately 52 weeks ]
    Part A

  2. Incidence of serious adverse events (SAEs) [ Time Frame: 12 months post-treatment, approximately 52 weeks ]
    Parts A and B

  3. Incidence and severity of treatment-emergent adverse events (TEAEs) up [ Time Frame: 12 months post-treatment, approximately 52 weeks ]
    Parts A and B

  4. Overall response rate (ORR) [ Time Frame: 6 months post-treatment, approximately 26 weeks ]
    Part B


Secondary Outcome Measures :
  1. ORR [ Time Frame: 3 months post-treatment, approximately 12 weeks ]
    Parts A and B

  2. Complete response (CR) [ Time Frame: 3 months post-treatment, approximately 12 weeks ]
    Parts A and B

  3. Partial response (PR) [ Time Frame: 3 months post-treatment, approximately 12 weeks ]
    Parts A and B

  4. Time to best response [ Time Frame: Up to 12 months ]
    Parts A and B

  5. Time to first response [ Time Frame: Up to 12 months ]
    Parts A and B

  6. Proportion of patients who maintain any clinical response [ Time Frame: Up to 12 months ]
    Parts A and B

  7. Platelet transfusions per month over time [ Time Frame: Up to 12 months ]
    Parts A and B

  8. Red blood cell transfusions per month over time [ Time Frame: Up to 12 months ]
    Parts A and B

  9. Changes in lymphocyte cell counts [ Time Frame: Up to 12 months ]
    Parts A and B

  10. Changes in neutrophil cell counts [ Time Frame: Up to 12 months ]
    Parts A and B

  11. Changes in hemoglobin cell counts [ Time Frame: Up to 12 months ]
    Parts A and B

  12. Changes in reticulocyte cell counts [ Time Frame: Up to 12 months ]
    Parts A and B

  13. Changes in platelet cell counts [ Time Frame: Up to 12 months ]
    Parts A and B

  14. Changes in the whole blood immune cell subsets (T cells) [ Time Frame: Up to 12 months ]
    Parts A and B

  15. Changes in the whole blood immune cell subsets (B cells) [ Time Frame: Up to 12 months ]
    Parts A and B

  16. Changes in the whole blood immune cell subsets (NK cells) [ Time Frame: Up to 12 months ]
    Parts A and B

  17. Drug concentrations in serum over time [ Time Frame: Up to 12 months ]
    Parts A and B

  18. Incidence of treatment-emergent anti-drug antibody (ADA) over time [ Time Frame: Up to 12 months ]
    Parts A and B


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • SAA that is IST-refractory or IST-relapsed, as defined in the protocol
  • Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
  • Adequate hepatic and renal function as defined in the protocol

Key Exclusion Criteria:

  • Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as defined in the protocol
  • Evidence of myelodysplastic syndrome as defined in the protocol
  • Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of clinically significant hemolysis (eg, treatment indicated) or history of PNH-associated thrombosis
  • Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
  • Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing as defined in the protocol
  • 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
  • HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit as defined in the protocol
  • Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI
  • Active infection as defined in the protocol including COVID-19

Note: Other protocol-defined inclusion/ exclusion criteria apply

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04409080


Contacts
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Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

Locations
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United States, Ohio
Cleveland Clinic Foundation Recruiting
Cleveland, Ohio, United States, 44195
Principal Investigator: Sophia Balderman         
United States, Texas
The University of Texas MD Anderson Cancer Center Not yet recruiting
Houston, Texas, United States, 77030
Principal Investigator: Tapan Kadia         
France
Hopital Saint-Louis - APHP Recruiting
Paris, Ile-de-France, France, 75010
Korea, Republic of
Gachon University Gil Hospital Recruiting
Incheon, Gyeonggi, Korea, Republic of, 21565
Principal Investigator: Hawk Kim         
Seoul National University Hospital Recruiting
Seoul, Seoul Capital Area, Korea, Republic of, 03080
Principal Investigator: Yoon Sung Soo         
Samsung Medical Center Recruiting
Seoul, Seoul Capital Area, Korea, Republic of, 06351
Principal Investigator: Jun Ho Yang         
The Catholic University of Korea, Seoul St. Marys Hospital Recruiting
Seoul, Seoul Capital Area, Korea, Republic of, 06591
Principal Investigator: Jong Wook Lee         
Ewha Womans University Medical Centre Recruiting
Seoul, Seoul Capital Area, Korea, Republic of, 07985
Principal Investigator: Yueng Chul Mun         
United Kingdom
King's College Hospital, London Recruiting
London, United Kingdom, SE5 9RS
Principal Investigator: Austin Kulasekararaj         
Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
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Study Director: Clinical Trial Management Regeneron Pharmaceuticals
More Information
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Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04409080    
Other Study ID Numbers: R7257-RAA-1947
2020-002031-29 ( EudraCT Number )
First Posted: June 1, 2020    Key Record Dates
Last Update Posted: June 27, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Analytic Code
Time Frame: Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
Access Criteria: Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency [EMA], Pharmaceuticals and Medical Devices Agency [PMDA], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
URL: https://vivli.org/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regeneron Pharmaceuticals:
Immunosuppressive therapy (IST)
Refractory
Relapsed
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases