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

Conditions

• Non-Transfusion Dependent Beta-Thalassemia (NTDT)

Eligibility Criteria

Sex

ALL

Age

18 Years - 65 Years

Healthy Volunteers

Not accepted

Interventions

• REGN7999 — DRUG
  Administered subcutaneous (SC)
• Placebo — DRUG
  Administered SC

Study Details

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)

Key Dates

Start date

Sep 30, 2024

Status verified

Mar 2026

Primary completion

Jan 24, 2028

Completion

Jan 24, 2028

Study Design

Enrollment

95 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Part A High Dose
  Randomized 2:2:1
• Experimental: Part A Low Dose
  Randomized 2:2:1
• Placebo Comparator: Part A Placebo
  Randomized 2:2:1
• Experimental: Part B High Dose
  Randomized 2:2:1
• Experimental: Part B Low Dose
  Randomized 2:2:1
• Placebo Comparator: Part B Placebo
  Randomized 2:2:1

Primary Outcome Measure

Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI) [ Time Frame: At week 24 ]

Central Contacts

• Clinical Trials Administrator
  844-734-6643
  [email protected]

Locations (1)

Find similar trials in New York, NY