REGN7999 for Beta Thalassemia

(FERVENT-1 Trial)

Recruiting at 20 trial locations

Overseen By Clinical Trials Administrator

Age: 18 - 65

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

Trial Summary

What is the purpose of this trial?

This study is researching an experimental drug called REGN7999 (called "study drug"). 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 study is looking at several other research questions, including: * Whether the study drug lowers extra iron levels in the body * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

Research Team

Clinical Trial Management

Principal Investigator

Regeneron Pharmaceuticals

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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