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Purpose:
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia (SAA) that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in IST-refractory/relapsed patients.
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
Status:
Recruiting
Bone Marrow Disease(s):
Phase:
Phase 1
Phase 2
Gender:
Female
Male
Age Group:
18 years and older
Accepts Healthy Volunteers:
No
Details on clinicaltrials.gov:
Inclusion Criteria:
- SAA that is refractory to or has relapsed while on standard of care IST, 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:
- Diagnosis of Fanconi anemia as defined in the protocol
- Evidence of myelodysplastic syndrome as defined in the protocol
- Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis 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
- 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
- Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI
Note: Other protocol-defined inclusion/ exclusion criteria apply.