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Testing a new antibody for treating iron overload in beta-thalassemia patients

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

Phase 2 Interventional Regeneron Pharmaceuticals · NCT06421636

This study is testing a new antibody called REGN7999 to see if it can help adults with beta-thalassemia lower their iron levels safely.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment95 (estimated)
Ages18 Years to 65 Years
SexAll
SponsorRegeneron Pharmaceuticals Industry-sponsored
Locations26 sites (New York, New York and 25 other locations)
Trial IDNCT06421636 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, tolerability, and efficacy of an experimental antibody called REGN7999 for adults with non-transfusion dependent beta-thalassemia. Participants will receive either the study drug or a placebo, and the study will measure iron levels in the body using MRI scans. The research aims to determine if REGN7999 can effectively lower excess iron levels and assess any potential side effects. Additionally, the study will monitor how the body responds to the drug over time.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with non-transfusion dependent beta-thalassemia who have elevated iron levels as indicated by MRI.

Not a fit: Patients with severe anemia, recent blood transfusions, or certain liver and kidney conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce iron overload in patients with non-transfusion dependent beta-thalassemia, improving their overall health and quality of life.

How similar studies have performed: While this approach is novel, similar studies targeting iron overload in beta-thalassemia have shown promise, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Clinical diagnosis of NTDT as described in the protocol
2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening
3. Serum ferritin ≥ 300 ng/mL as described in the protocol

Key Exclusion Criteria:

1. Hemoglobin ≤ 8 g/dL at screening
2. Any RBC transfusion within 12 weeks of visit 3
3. For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
4. For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol
5. Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
6. Absolute contraindication to MRI
7. Diagnosis of cirrhosis of the liver
8. Diagnosis of Chronic kidney disease (CKD) stage 4 or higher

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Where this trial is running

New York, New York and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Non-Transfusion Dependent Beta-ThalassemiaBeta-ThalassemiaResultant Iron Overload
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.