Natural History in Subjects with Fibrodysplasia Ossificans Progressiva
Research type
Research Study
Full title
A Natural History, Non-Interventional, Two-Part Study in Subjects with Fibrodysplasia Ossificans Progressiva (FOP). Study Number: PVO-1A-001
IRAS ID
160742
Contact name
Richard Keen
Contact email
Sponsor organisation
Clementia Pharmaceuticals Inc.
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 8 months, 18 days
Research summary
This is a multi-center, natural history, non-interventional, longitudinal, two-part study in subjects with classical FOP. This study will follow subjects for up to 36 months and is designed to gain insight into disease progression, impact on
subjects’ physical function, and assessment of clinical features and biomarkers that may be useful in diagnosis, monitoring disease progression, and potential treatment effects in subsequent interventional trials.
The study has been conducted in two parts, Part A and Part B.
• In Part A, up to 10 subjects ≥18 years of age have been enrolled. After subjects have completed the baseline imaging utilizing low-dose whole body CT scan(excluding head) and DEXA scan, the images have been analyzed by the imaging
committee to determine which imaging modality provides the optimal method
for assessing total body HO (heterotopic ossification). The optimal modality has been based on the ability of the modality to assess HO balanced against the estimated radiation exposure.
• In Part B, 104 additional subjects have been enrolled (19 subjects were enrolled in Clementia interventionnel studies and withdrew the NHS) , with
baseline imaging performed using the optimal modality. The optimal imaging
modality from Part A is then used to evaluate disease progression as
described above. It has been determined in Part A that whole body, low-dose CT
scan (excluding head) is the optimal imaging modality, it is performed at screening/baseline and annual study visits (Study Month 12, 24, and 36) or early termination.Up to 100 subjects subjects has been enrolled in total at 7 centers (USA, UK, Argentina, Australia, Italy and France).
REC name
London - Stanmore Research Ethics Committee
REC reference
15/LO/0630
Date of REC Opinion
30 Jun 2015
REC opinion
Further Information Favourable Opinion