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 Information
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
Administrative Informations
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