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

Brief Title

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

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

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
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Primary Outcome

Incidence of adverse events (AEs)

Secondary Outcome

 ORR

Condition

Severe Aplastic Anemia (SAA)

Intervention

REGN7257

Study Arms / Comparison Groups

 Part A and Part B

Description:  Part A: Single ascending dose Part B: Preferred dose

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

57

Start Date

January 13, 2021

Completion Date

August 8, 2024

Primary Completion Date

August 8, 2024

Eligibility 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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Clinical Trial Management, 844-734-6643, [email protected]

Location Countries

France

Location Countries

France

NCT ID

NCT04409080

Organization ID

R7257-RAA-1947

Secondary IDs

2020-002031-29

Responsible Party

Sponsor

Study Sponsor

Regeneron Pharmaceuticals

Study Sponsor

Clinical Trial Management, Study Director, Regeneron Pharmaceuticals

Verification Date

June 2022