RECRUITING

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

Description

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)

Conditions

Non-transfusion Dependent Beta-thalassemia (NTDT)
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Related Conditions:

Non-transfusion Dependent Beta-thalassemia (NTDT)

Study Overview

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Study Details

Study 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)

A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA

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

Condition
Non-transfusion Dependent Beta-thalassemia (NTDT)
Intervention / Treatment

-

Contacts and Locations

New York

Weill Cornell Medicine, New York, New York, United States, 10065

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • 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

Ages Eligible for Study

18 Years to 60 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Regeneron Pharmaceuticals,

Clinical Trial Management, STUDY_DIRECTOR, Regeneron Pharmaceuticals

Study Record Dates

2028-01-24