REGN7257 in Adult Patients with Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
- Conditions
- Severe aplastic anemia (SAA)
- Registration Number
- 2023-508601-24-00
- Lead Sponsor
- Regeneron Pharmaceuticals Inc.
- Brief Summary
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with IST-refractory or IST-relapsed SAA. An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in patients with IST-relapsed SAA as proof of concept
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ended
- Sex
- Not specified
- Target Recruitment
- 14
Part A: SAA that is IST-refractory or IST-relapsed, as defined in the protocol
Part B: SAA that is 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
Other protocol-defined inclusion criteria apply
Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as defined in the protocol
Other protocol-defined exclusion criteria apply
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 for patients enrolled in Part A
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Part A: Incidence of adverse events (AEs), incidence of serious adverse events (SAEs), and incidence and severity of treatment-emergent adverse events (TEAEs); Part A: Incidence of adverse events (AEs), incidence of serious adverse events (SAEs), and incidence and severity of treatment-emergent adverse events (TEAEs);
Part B: Incidence of serious adverse events (SAEs), incidence and severity of treatment-emergent adverse events (TEAEs); Part B: Incidence of serious adverse events (SAEs), incidence and severity of treatment-emergent adverse events (TEAEs);
Part B: Overall response rate (ORR); Part B: Overall response rate (ORR);
- Secondary Outcome Measures
Name Time Method Any clinical response; Part A Any clinical response; Part A
Any clinical response; Part B Any clinical response; Part B
Overall response rate (ORR); Parts A & B Overall response rate (ORR); Parts A & B
Complete response (CR); Parts A and B Complete response (CR); Parts A and B
Partial response (PR); Parts A and B Partial response (PR); Parts A and B
Time to best response; Part A Time to best response; Part A
Time to best response; Part B Time to best response; Part B
Time to first response; Part A Time to first response; Part A
Time to first response; Part B Time to first response; Part B
Platelet transfusions per month over time; Part A Platelet transfusions per month over time; Part A
Platelet transfusions per month over time; Part B Platelet transfusions per month over time; Part B
Red blood cell transfusions per month over time; Part A Red blood cell transfusions per month over time; Part A
Red blood cell transfusions per month over time; Part B Red blood cell transfusions per month over time; Part B
Changes in lymphocyte cell counts; Part A Changes in lymphocyte cell counts; Part A
Changes in lymphocyte cell counts; Part B Changes in lymphocyte cell counts; Part B
Changes in neutrophil cell counts; Part A Changes in neutrophil cell counts; Part A
Changes in neutrophil cell counts; Part B Changes in neutrophil cell counts; Part B
Changes in hemoglobin cell counts; Part A Changes in hemoglobin cell counts; Part A
Changes in hemoglobin cell counts; Part B Changes in hemoglobin cell counts; Part B
Changes in reticulocyte cell counts; Part A Changes in reticulocyte cell counts; Part A
Changes in reticulocyte cell counts; Part B Changes in reticulocyte cell counts; Part B
Changes in platelet cell counts; Part A Changes in platelet cell counts; Part A
Changes in platelet cell counts; Part B Changes in platelet cell counts; Part B
Changes in the whole blood immune cell subsets (T cells); Part A Changes in the whole blood immune cell subsets (T cells); Part A
Changes in the whole blood immune cell subsets (T cells); Part B Changes in the whole blood immune cell subsets (T cells); Part B
Changes in the whole blood immune cell subsets (B cells); Part A Changes in the whole blood immune cell subsets (B cells); Part A
Changes in the whole blood immune cell subsets (B cells); Part B Changes in the whole blood immune cell subsets (B cells); Part B
Changes in the whole blood immune cell subsets [Natural killer (NK) cells]; Part A Changes in the whole blood immune cell subsets [Natural killer (NK) cells]; Part A
Changes in the whole blood immune cell subsets (NK cells); Part B Changes in the whole blood immune cell subsets (NK cells); Part B
Drug concentrations in serum over time; Part A Drug concentrations in serum over time; Part A
Drug concentrations in serum over time; Part B Drug concentrations in serum over time; Part B
Incidence of treatment-emergent anti-drug antibody (ADA) over time; Part A Incidence of treatment-emergent anti-drug antibody (ADA) over time; Part A
Incidence of treatment-emergent ADA over time; Part B Incidence of treatment-emergent ADA over time; Part B
Trial Locations
- Locations (1)
Hopital Saint Louis
🇫🇷Paris, France
Hopital Saint Louis🇫🇷Paris, FranceRegis Peffault de LatourSite contact+33142499213regis.peffaultdelatour@aphp.fr