NCT04409080

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

A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 \[ADA\])

Detailed description

The trial was intended to be a Phase 1/2 trial, but no participants were enrolled in Phase 2

Conditions

• Severe Aplastic Anemia (SAA)

Interventions

Type	Name	Description
DRUG	REGN7257	Administered by intravenous (IV) infusion, in Part A and B.

Timeline

• Start date: 2021-01-13
• Primary completion: 2024-10-17
• Completion: 2024-10-17
• First posted: 2020-06-01
• Last updated: 2025-10-24

Locations

10 sites across 4 countries: United States, France, South Korea, United Kingdom

Regulatory

• FDA-regulated drug study