NCT06421636

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 95 (estimated)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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)

Conditions

• Non-Transfusion Dependent Beta-Thalassemia (NTDT)

Interventions

Type	Name	Description
DRUG	REGN7999	Administered subcutaneous (SC)
DRUG	Placebo	Administered SC

Timeline

• Start date: 2024-09-30
• Primary completion: 2028-01-24
• Completion: 2028-01-24
• First posted: 2024-05-20
• Last updated: 2026-03-18

Locations

26 sites across 9 countries: United States, Georgia, Greece, India, Italy, Malaysia, Thailand, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study