REGN7999 for Beta Thalassemia

(FERVENT-1 Trial)

Recruiting at 27 trial locations

Overseen By Clinical Trials Administrator

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Regeneron Pharmaceuticals

Must not be taking: Luspatercept, Mitapivat

Disqualifiers: Cirrhosis, CKD stage 4, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 12 weeks

95 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called REGN7999, a potential drug for individuals with non-transfusion dependent beta-thalassemia, a blood disorder. The primary goal is to determine the treatment's safety and effectiveness, particularly in reducing excess iron levels in the body. Researchers will also monitor for side effects and assess the body's response to the drug over time. Eligible participants should have beta-thalassemia and high iron levels but should not have received a recent blood transfusion. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial requires that you have not used certain medications like luspatercept or mitapivat in the 6 months before screening. If you are on Iron Chelation Therapy, you must not change the dose in the 12 weeks before screening. The protocol does not specify other medication restrictions, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that REGN7999 is likely to be safe for humans?

Research has shown that REGN7999 was well-tolerated in earlier studies. In one study, healthy volunteers took single doses of REGN7999 and did not experience any major side effects. This study also found that the drug effectively lowered iron levels in the blood without causing serious problems.

Additionally, animal studies demonstrated that REGN7999 successfully reduced iron levels in the liver, which is important for treating conditions like beta-thalassemia. While these results are encouraging, it's important to remember that this treatment is still under investigation. Researchers are closely monitoring for any side effects as they gather more information on its safety and effectiveness for people with beta-thalassemia. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for beta-thalassemia?

Researchers are excited about REGN7999 for beta thalassemia because it offers a novel approach compared to standard treatments like blood transfusions and iron chelation therapy. Unlike these methods, which manage symptoms, REGN7999 is designed to target the underlying issues by enhancing erythropoiesis, the process of producing red blood cells. This new mechanism could potentially reduce the need for frequent transfusions and improve overall quality of life for patients. Additionally, this approach might address complications related to iron overload, a common problem with traditional treatments.

What evidence suggests that REGN7999 might be an effective treatment for beta thalassemia?

Earlier research has shown that REGN7999 holds promise for treating non-transfusion dependent beta-thalassemia. Studies suggest that this drug blocks a protein called TMPRSS6, which helps reduce excess iron in the body and improves red blood cell function. One study found that patients who received single doses of REGN7999 experienced a noticeable increase in hepcidin, a protein that controls iron levels. These findings indicate that REGN7999 might effectively manage iron overload and improve symptoms for people with beta-thalassemia. Participants in this trial will receive different doses of REGN7999 or a placebo to further evaluate its effectiveness and safety. ^{1 2 3 4 6}

Who Is on the Research Team?

Clinical Trial Management

Principal Investigator

Regeneron Pharmaceuticals

Are You a Good Fit for This Trial?

Adults with non-transfusion dependent beta-thalassemia and iron overload can join this trial. They must have an iron level in the liver of at least 5 mg Fe/g dry weight, as shown by MRI, and a serum ferritin level of 300 ng/mL or more.

Inclusion Criteria

My serum ferritin level is 300 ng/mL or higher.

My liver iron concentration is high, as confirmed by an MRI.

I have been diagnosed with non-transfusion-dependent thalassemia.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous injections of REGN7999 or placebo to evaluate efficacy, safety, and tolerability

12 weeks

6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• REGN7999

Trial Overview The trial is testing REGN7999, an experimental antibody for treating iron overload. Participants will receive injections under the skin. The study compares the effects of REGN7999 to a placebo while monitoring iron levels using MRI scans.

How Is the Trial Designed?

Group I: Part B Low Dose Experimental Treatment 1 Intervention

Randomized 2:2:1

Group II: Part B High Dose Experimental Treatment 1 Intervention

Randomized 2:2:1

Group III: Part A Low Dose Experimental Treatment 1 Intervention

Randomized 2:2:1

Group IV: Part A High Dose Experimental Treatment 1 Intervention

Randomized 2:2:1

Group V: Part A Placebo Placebo Group 1 Intervention

Randomized 2:2:1

Group VI: Part B Placebo Placebo Group 1 Intervention

Randomized 2:2:1

Who Is Running the Clinical Trial?

Regeneron Pharmaceuticals

Lead Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06421636?term=REGN7999%0A%20Iron%20overload%20in%20beta-thalassemia&rank=1

A Study to Test the Safety, Tolerability, and Efficacy of ... The study is focused on patients with non-transfusion dependent beta - thalassemia . The aim of the study is to see how safe and effective the study drug is.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12220967/

A TMPRSS6-inhibiting mAb improves disease in a β ... Our results suggest that, by both reducing iron and improving RBC function, inhibition of TMPRSS6 by REGN7999 may offer a therapy for iron ...

3. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/3841/502364/Single-Ascending-Doses-of-REGN7999-a-Monoclonal

Single Ascending Doses of REGN7999, a Monoclonal ... Single doses of REGN7999 (ranging from 10 to 900 mg) resulted in acute 2-fold to 12-fold increases in serum hepcidin among all the REGN7999 ...

4. clinicaltrials.regeneron.com clinicaltrials.regeneron.com/clinical-trials/a0Mdy0000004ajFEAQ/nct06421636

REGENERON - Clinical Trials The study is focused on patients with non-transfusion dependent beta-thalassemia. The aim of the study is to see how safe and effective the study drug is. The ...

5. library.ehaweb.org library.ehaweb.org/eha/2025/eha2025-congress/4162614/nikhil.singh.efficacy.safety.and.tolerability.of.regn7999.in.patients.with.html?f=listing%3D3%2Abrowseby%3D8%2Asortby%3D1%2Amedia%3D1

EFFICACY, SAFETY, AND TOLERABILITY OF REGN7999 IN ... Summary/Conclusion: This study will assess the efficacy, safety, and tolerability of REGN7999 in patients with IOL due to non-transfusion- ...

6. clinicaltrials.eu clinicaltrials.eu/inn/regn7999/

REGN7999 – Application in Therapy and Current Clinical ... REGN7999 is currently being tested in healthy volunteers to establish its safety profile and understand how it works in the body. It's also being studied in ...