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

NCT ID: NCT04409080

Last Updated: 2025-10-24

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-01-13
• Study Completion Date: 2024-10-17

Brief 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)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Part A

Part A: Single ascending dose (SAD) escalation cohorts

Group Type: EXPERIMENTAL

REGN7257

Intervention Type: DRUG

Administered by intravenous (IV) infusion, in Part A and B.

Part B

Part B: Multiple REGN7257 dosages.

Group Type: EXPERIMENTAL

REGN7257

Intervention Type: DRUG

Administered by intravenous (IV) infusion, in Part A and B.

Interventions

REGN7257

Administered by intravenous (IV) infusion, in Part A and B.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Part A: SAA that is IST-refractory or IST-relapsed, as defined in the protocol

2. Part B: SAA that is IST-relapsed, as defined in the protocol

3. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient

4. Adequate hepatic and renal function as defined in the protocol

Exclusion Criteria

1. Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as defined in the protocol

2. Evidence of myelodysplastic syndrome as defined in the protocol

3. Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of clinically significant hemolysis (eg, treatment indicated) 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 for patients enrolled in Part A

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 as defined in the protocol

8. Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI

9. Active infection as defined in the protocol

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

Cleveland Clinic Foundation

Cleveland, Ohio, United States

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Hopital Saint-Louis - APHP

Paris, Île-de-France Region, France

Gachon University Gil Hospital

Incheon, Gyeonggi-do, South Korea

Seoul National University Hospital

Seoul, Seoul Capital Area, South Korea

Samsung Medical Center

Seoul, Seoul Capital Area, South Korea

The Catholic University of Korea, Seoul St. Marys Hospital

Seoul, Seoul Capital Area, South Korea

Ewha Womans University Medical Centre

Seoul, Seoul Capital Area, South Korea

St James's University Hospital

Leeds, West Yorkshire, United Kingdom

King's College Hospital, London

London, , United Kingdom

Countries

United States; France; South Korea; United Kingdom

Other Identifiers

2020-002031-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-508601-24-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

R7257-RAA-1947

Identifier Type: -

Identifier Source: org_study_id