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

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Conditions

Non-transfusion Dependent Beta-thalassemia (NTDT)Beta-thalassemiaResultant Iron overload (IOL)

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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)

Official Title

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

Quick Facts

Study Start:2024-09-30
Study Completion:2028-01-24
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06421636

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.

Ages Eligible for Study:18 Years to 60 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Clinical diagnosis of NTDT as described in the protocol
  2. 2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening
  3. 3. Serum ferritin ≥ 300 ng/mL as described in the protocol
  1. 1. Hemoglobin ≤ 8 g/dL
  2. 2. Any RBC transfusion within 12 weeks of visit 3
  3. 3. For Part A only: Any ICT use in approximately 12 weeks prior to screening as described in the protocol
  4. 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. 5. Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
  6. 6. Absolute contraindication to MRI
  7. 7. Diagnosis of cirrhosis of the liver
  8. 8. Diagnosis of Chronic kidney disease (CKD) stage 4 or higher

Contacts and Locations

Study Contact

Clinical Trials Administrator
CONTACT
844-734-6643
clinicaltrials@regeneron.com

Principal Investigator

Clinical Trial Management
STUDY_DIRECTOR
Regeneron Pharmaceuticals

Study Locations (Sites)

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

Collaborators and Investigators

Sponsor: Regeneron Pharmaceuticals

  • Clinical Trial Management, STUDY_DIRECTOR, Regeneron Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-30
Study Completion Date2028-01-24

Study Record Updates

Study Start Date2024-09-30
Study Completion Date2028-01-24

Terms related to this study

Keywords Provided by Researchers

  • Beta-thalassemia
  • Resultant Iron overload (IOL)

Additional Relevant MeSH Terms

  • Non-transfusion Dependent Beta-thalassemia (NTDT)