A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under t… (NCT06421636) | Clinical Trial Compass
RecruitingPhase 2
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
United States, Georgia, Greece95 participantsStarted 2024-09-30
Plain-language 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)
Who can participate
Age range18 Years – 65 Years
SexALL
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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
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
What they're measuring
1
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
Timeframe: At week 24
2
Incidence of Treatment-Emergent Adverse Events (TEAEs)