Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Conditions
Keywords
Beta-Thalassemia, Resultant Iron Overload (IOL)
Brief 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)
Interventions
DRUGREGN7999
Administered subcutaneous (SC)
DRUGPlacebo
Administered SC
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI) | At week 24 |
| Incidence of Treatment-Emergent Adverse Events (TEAEs) | Up to week 72 |
| Severity of TEAEs | Up to week 72 |
Secondary
| Measure | Time frame |
|---|---|
| Achievement of ≥20% reduction from baseline in LIC by R2* MRI | Through week 52 |
| Change from baseline in hemoglobin | To week 24 |
| Change from baseline in LIC by R2* MRI | At week 52 |
| Percent change from baseline in LIC by R2* MRI | To week 24 and week 52 |
| Change in hemoglobin over time | Up to week 56 |
| Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of Red Blood Cell (RBC) transfusions | Baseline to week 56 |
| Number of RBC transfusions required | Baseline to week 72 |
| Achievement of transfusion independence | Baseline to week 72 |
| Change in RBC counts over time | Baseline to week 56 |
| Concentrations of REGN7999 in serum over time | Up to week 56 |
| Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time | Up to week 56 |
| Magnitude of ADA to REGN7999 over time | Up to week 56 |
Countries
Georgia, Greece, India, Italy, Malaysia, Thailand, Turkey (Türkiye), United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed