TerminatedPhase 1

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

Stopped: Sponsor Decision

United States, France, South Korea17 participantsStarted 2021-01-13

Plain-language summary

This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the production of blood cells. The main purpose of this two-part study (Part A and Part B) is to test how safe and tolerable REGN7257 is in patients with SAA in which other Immunosuppressive therapies (ISTs) have not worked well. The study is looking at several other research questions to better understand the following properties of REGN7257: * Side effects that may be experienced by participants taking REGN7257 * How REGN7257 works in the body * How much REGN7257 is present in blood after dosing * If REGN7257 works to raise levels of certain blood counts after treatment * How quickly REGN7257 works to raise levels of certain blood counts * In patients for whom REGN7257 works to raise levels of certain blood counts after treatment, how many continue to show such a response throughout the study * If REGN7257 works to lower the number of platelet and red blood cell transfusions needed * How REGN7257 changes immune cell counts and composition * How the body reacts to REGN7257 and if it produces proteins that bind to REGN7257 (this would be called the formation of anti-drug antibodies \[ADA\])

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

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 for patients enrolled in Part A

What they're measuring

Incidence of adverse events (AEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Timeframe: Through the end of study visit, approximately 78 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: Through the end of study visit, approximately 78 weeks

Overall response rate (ORR)

Timeframe: At 6 months, approximately 26 weeks

Trial details

NCT IDNCT04409080

SponsorRegeneron Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-10-17

View on ClinicalTrials.gov More Severe Aplastic Anemia (SAA) trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

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 for patients enrolled in Part A

What they're measuring

Incidence of adverse events (AEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Timeframe: Through the end of study visit, approximately 78 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Timeframe: Through the end of study visit, approximately 78 weeks

Overall response rate (ORR)

Timeframe: At 6 months, approximately 26 weeks