A Phase II study of the effects of REGN2477 in patients with FOP
Research type
Research Study
Full title
A randomised, placebo-controlled study to assess the safety, tolerability, pharmacokinetics and effects on heterotopic bone formation of REGN2477 in patients with fibrodysplasia ossificans progressiva
IRAS ID
225341
Contact name
Richard Keen
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2016-005035-33
Duration of Study in the UK
2 years, 5 months, 0 days
Research summary
This study is a phase II study for patients diagnosed with Fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder in which bone forms in muscles, tendons, and other connective tissue. This is called heterotopic ossification or “HO” because the bone formation is in abnormal locations.The study involves a study drug,REGN2477 which is a targeted drug that may inhibits (HO)in patients with FOP.
The study is a randomised, placebo controlled study with up to 40 patients with FOP enrolled at multiple sites globally.
This study has four periods:
Screening/Baseline Period Up to 4 weeks
Screening is the time when the study doctor determines eligibility to participate.
If patient is eligible to participate, procedures will be done to collect baseline information before the study treatment starts.
This period may involve multiple visits to the study site.
Double-Blind Treatment (Period 1) duration about 6 months.During this period, patients will be assigned by chance (randomised) and will receive either the REGN2477 or placebo(looks like the REGN2477 but it does not contain any active ingredient).Patient and study doctor will not know what study patient is receiving. There will be 9 visits in total during this period, including 3 visits in the first week (day 1, 2 and 7). After that, visits will be about every 4 weeks.
Open-Label REGN2477 Treatment (Period 2)About 6 months.During this period, patient will receive actual REGN2477 (no placebo).Patient and study doctor both know that study patient is receiving REGN2477.There will be 7 visits in total during this period, about every 4 weeks.
Follow-up Period About 6 months from the last study treatment
There will be 6 visits in total during this period,every 4 weeks.
The follow-up period is after the treatment period, and is when study doctor monitors health without giving any more REGN2477 treatment.REC name
London - Central Research Ethics Committee
REC reference
17/LO/1016
Date of REC Opinion
10 Aug 2017
REC opinion
Further Information Favourable Opinion