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

About the study

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

Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION 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

EXCLUSION CRITERIA

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