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
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
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugREGN7999
Administered subcutaneous (SC)
- DrugPlacebo
Administered SC
Locations (26)
- Weill Cornell MedicineNew York, New York
- K Eristavi National Center of Experimental and Clinical SurgeryTbilisi
- Medinvest Institute of Hematology and Transfusiology LimitedTbilisi
- Children's Hospital Agia SophiaAthens, Attica
- Laiko General Hospital of AthensAthens, Attica
- University Hospital of IoanninaIoannina