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
Regeneron Pharmaceuticals
95 participants
Sep 30, 2024
INTERVENTIONAL
Conditions
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)
Eligibility
Inclusion Criteria3
- Clinical diagnosis of NTDT as described in the protocol
- IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening
- Serum ferritin ≥ 300 ng/mL as described in the protocol
Exclusion Criteria8
- Hemoglobin ≤ 8 g/dL at screening
- Any RBC transfusion within 12 weeks of visit 3
- For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
- For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol
- Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
- Absolute contraindication to MRI
- Diagnosis of cirrhosis of the liver
- Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
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Interventions
Administered subcutaneous (SC)
Administered SC
Locations(26)
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NCT06421636