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

Launched by REGENERON PHARMACEUTICALS · May 14, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new experimental drug called REGN7999, which is being injected under the skin to treat adults with non-transfusion dependent beta-thalassemia (NTDT). The main goal of the study is to find out how safe and effective this drug is in helping lower excess iron levels in the body, as iron overload can be a serious issue for people with NTDT. Researchers will use MRI scans to measure the iron levels in participants' bodies and will also monitor any side effects, how much of the drug is in the blood at different times, and whether the body develops antibodies against the drug, which could affect its effectiveness.

To participate in this trial, individuals must be diagnosed with NTDT and have a certain level of iron overload, as defined in the study's protocol. Key requirements include having a specific measurement of iron in their liver and a serum ferritin level of at least 300 ng/mL. However, patients with very low hemoglobin levels, recent blood transfusions, or certain other health conditions will not be eligible. If you decide to join the study, you'll be closely monitored by healthcare professionals throughout the trial to ensure your safety and to gather important information about how well the drug works.

Gender

ALL

Eligibility 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
• • 2. Any RBC transfusion within 12 weeks of visit 3
• • 3. For Part A only: Any 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 Iron chelation therapy (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/ Exclusion Criteria apply

Trial Officials