REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
Aplastic Anemia / Posted 10 months ago
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
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Inclusion Criteria :
- Ages Eligible for Study: 18 Years and older
- Sexes Eligible for Study: All
- Accepts Healthy Volunteers: No
- 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
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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
- Study end date : August 8, 2024
- Wales-Based Study Contact : Please speak to your clinician
- Principal Investigator : Austin Kulasekararaj (King's College London)