Phase 2 safety, tolerability & efficacy study of subcut REGN7999 in participants with β-thalassemia
Research type
Research Study
Full 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
IRAS ID
1010143
Contact name
Mikeal Paiyz
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
This is a phase 2, randomized, double-blind, placebo-controlled trial. Randomized means that the treatment each participant receives will be randomly assigned by a computer program. Double-blind means none of the participants, researchers, trial doctors, or other trial staff will know which treatment each participant is receiving. Placebo-controlled means that some participants will receive a placebo. The placebo will look like the study drug (REGN7999) but will not have any active REGN7999 in it. After a screening period of up to 6 weeks, each participant will be in the trial for about 72 weeks. This trial will have 2 parts. Part A will include up to about 25 participants and Part B will include about 70 participants. All participants will be 18 to 60 years of age and have iron overload due to Non-Transfusion Dependent β-Thalassemia. Participants in Part A must not be taking any chelators for at least 12 weeks before joining the trial. Participants in Part B must either not be taking any chelators or be taking a dose that has not changed for at least 12 weeks before joining the trial. People cannot join the trial if they have any long-term or serious medical condition that would make it too risky or difficult to participate. Depending on when participants join the trial, they will either be in Part A or Part B, but not both. Part B will begin after all of the participants in Part A have had their first visit. In Part A, participants will receive either one of 2 doses of REGN7999 or placebo. For Part B, researchers will decide on a “high dose” and a “lower dose” of REGN7999 based on some of the results from Part A. The trial is primarily investigating whether REGN7999 reduces the amount of iron in participants’ livers. Researchers will measure the amount of iron in participants’ livers using a type of MRI scan. Researchers will look at the change in the amount of iron in participants’ livers at their first visit (baseline) compared to the amount at 24 weeks.
REC name
North of Scotland Research Ethics Committee 1
REC reference
24/NS/0070
Date of REC Opinion
13 Aug 2024
REC opinion
Further Information Favourable Opinion