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

Phase 1

• Conditions: SEVERE APLASTIC ANEMIA; MedDRA version: 20.0Level: LLTClassification code 10002274Term: Anemia aplasticSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2020-002031-29-FR
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-07-10
• Last Update Posted: 5 January 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

1. SAA that is refractory to or has relapsed while on standard of care IST, as defined in the protocol
2. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
3. Adequate hepatic and renal function as defined in the protocol

Other protocol-defined inclusion criteria apply
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 24

Exclusion Criteria

1. Diagnosis of Fanconi anemia as defined in the protocol
2. Evidence of myelodysplastic syndrome as defined in the protocol
3. Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis or history of PNH-associated thrombosis
4. Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
5. Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing
6. 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
7. HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit
8. Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI

Other protocol-defined exclusion criteria apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified