What is the mechanism of action of this treatment?

Immune modulation therapies, such as antithymocyte globulin (ATG) and cyclosporine, are common treatments for Aplastic Anemia. These therapies work by suppressing the immune system, which is believed to attack the bone marrow in this condition. By mitigating this immune-mediated damage, these treatments help restore the bone marrow's ability to produce blood cells. This is vital for patients as it can reduce the need for transfusions, improve blood counts, and enhance overall quality of life.

What side effects are associated with this type of intervention?

Common treatments for aplastic anemia, particularly those involving immune modulation such as immunosuppressive therapies (ISTs), can have several side effects. These may include increased susceptibility to infections due to suppressed immune function, liver toxicity, and gastrointestinal disturbances. Specific drugs like cyclosporine and antithymocyte globulin (ATG) can cause renal dysfunction, hypertension, and infusion-related reactions. Additionally, long-term use of these therapies can lead to an increased risk of secondary malignancies. The experimental drug REGN7257, being studied for its immune-modulating properties, is also being evaluated for similar side effects, including potential changes in immune cell counts and the formation of anti-drug antibodies.

What prior FDA approvals exist?

FDA-approved treatments for Aplastic Anemia often involve immune modulation to improve blood cell production. Key therapies include antithymocyte globulin (ATG) and cyclosporine, which suppress the immune system to allow bone marrow recovery. Eltrombopag, a thrombopoietin receptor agonist, has also been approved to stimulate platelet production in patients with insufficient response to immunosuppressive therapy. These treatments aim to enhance the body's ability to produce blood cells, similar to the investigational approach of REGN7257.

What other types of research exist?

Promising alternatives to REGN7257 for aplastic anemia patients include eltrombopag and romiplostim, which are thrombopoietin receptor agonists that stimulate platelet production. Additionally, avatrombopag and hetrombopag are newer thrombopoietin receptor agonists that have shown efficacy in clinical trials. These agents offer potential benefits in improving blood cell production and reducing the need for transfusions.

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Monoclonal Antibodies

REGN7257 for Severe Aplastic Anemia

Phase 1 & 2

Recruiting

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

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

Must not have

Diagnosis of Fanconi anemia or other congenital bone marrow failure 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months post-treatment, approximately 52 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called REGN7257 for patients with severe aplastic anemia who haven't responded to other treatments. The drug aims to help the bone marrow produce more blood cells and reduce the need for transfusions. The study will check if the drug is safe and how well it works.

Who is the study for?

This trial is for adults with severe aplastic anemia who haven't improved or have relapsed after immunosuppressive therapy. They can't be candidates for stem cell transplant, either because it's not suitable or they've refused it. Participants need to have good liver and kidney function.

What is being tested?

The study tests REGN7257's safety and how well patients tolerate it. For part of the study, they'll also check if the drug effectively treats severe aplastic anemia by observing clinical responses, changes in transfusion needs, blood counts, and immune reactions over time.

What are the potential side effects?

While specific side effects of REGN7257 are not listed here, common ones may include reactions at the injection site, fatigue, headache, nausea and potential impacts on liver enzymes which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I cannot or do not want to undergo a stem cell transplant.

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My SAA hasn't responded to or has returned after standard treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with Fanconi anemia or another similar bone marrow failure condition.

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I have PNH with significant red blood cell breakdown or a history of blood clots due to PNH.

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I have been diagnosed with a bone marrow disorder.

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I have or had tuberculosis that was not fully treated.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months post-treatment, approximately 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months post-treatment, approximately 52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months post-treatment, approximately 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Incidence of adverse events (AEs)

Incidence of serious adverse events (SAEs)

+1 more

Secondary study objectives

Any clinical response

Changes in hemoglobin cell counts

Changes in lymphocyte cell counts

+17 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part BExperimental Treatment1 Intervention

Part B: Multiple REGN7257 dosages.

Group II: Part AExperimental Treatment1 Intervention

Part A: Single ascending dose (SAD) escalation cohorts

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Immune modulation therapies, such as antithymocyte globulin (ATG) and cyclosporine, are common treatments for Aplastic Anemia. These therapies work by suppressing the immune system, which is believed to attack the bone marrow in this condition. By mitigating this immune-mediated damage, these treatments help restore the bone marrow's ability to produce blood cells. This is vital for patients as it can reduce the need for transfusions, improve blood counts, and enhance overall quality of life.