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

 Aplastic Anemia / Posted 2 weeks 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
  • 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
  • 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)
Contact details

King's College Hospital,London,England,SE5 9RS  Show Phone Number clinicaltrials@regeneron.com www.kch.nhs.uk

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