Garetosmab in Fibrodysplasia Ossificans Progressiva
Research type
Research Study
Full title
Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability, and Efficacy of Garetosmab in Patients with Fibrodysplasia Ossificans Progressiva
IRAS ID
1005772
Contact name
Edward Cox
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2022-000880-40
Research summary
The study is researching an experimental drug called garetosmab. The study is focused on adult participants with fibrodysplasia ossificans progressiva (FOP). The aim of the study is to see how safe and effective the study drug is in participants with FOP.
FOP is a rare disease in which bone forms in muscle, tendons, and other connective tissues.
This study will include about 66 people worldwide and has three treatment groups; two treatment groups of different doses of garetosmab given every 4 weeks and one treament group of placebo given every 4 weeks.
Participants will have a 1 in 3 chance of being assigned to any of these groups and will receive up to 14 doses of study drug or placebo. The study drug will be administered into a vein using a needle. This is called an intravenous (IV) infusion.
This study consists of three periods: a screening period, a treatment period, and a follow-up period. Overall, participation in this study will last about three years (from screening to end of study) and will involve up to 18 on-site visits, as well as a phone contact or remote end of study visit. Assessments to be performed include collection of vital signs, electrocardiograms (ECGs), blood and urine samples, computed tomography (CT) scans and lung function tests.REC name
South Central - Berkshire Research Ethics Committee
REC reference
22/SC/0209
Date of REC Opinion
19 Jul 2022
REC opinion
Further Information Favourable Opinion