Overview
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
Key Inclusion Criteria:
- 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
Key Exclusion Criteria:
- 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
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply