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Experimental drug for rare bone marrow disease shows early promise, but study halted

NCT ID NCT04409080

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental drug called REGN7257 in 17 people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The drug was given to patients whose disease did not get better or came back after standard immune-suppressing treatments. The main goal was to check safety and see if the drug could improve blood cell counts, but the study was stopped early.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

17 people

The number who actually took part.

Started

Jan 2021

Finished

Oct 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key 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 Key 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 Note: Other protocol-defined inclusion/ exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Severe aplastic anemia (SAA)

Contacts and locations

Locations

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Ewha Womans University Medical Centre

    Seoul, Seoul Capital Area, 07985, South Korea

  • Gachon University Gil Hospital

    Incheon, Gyeonggi-do, 21565, South Korea

  • Hopital Saint-Louis - APHP

    Paris, Île-de-France Region, 75010, France

  • King's College Hospital, London

    London, SE5 9RS, United Kingdom

  • Samsung Medical Center

    Seoul, Seoul Capital Area, 06351, South Korea

  • Seoul National University Hospital

    Seoul, Seoul Capital Area, 03080, South Korea

  • St James's University Hospital

    Leeds, West Yorkshire, LS97TF, United Kingdom

  • The Catholic University of Korea, Seoul St. Marys Hospital

    Seoul, Seoul Capital Area, 06591, South Korea

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.