Status:
COMPLETED
An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Lead Sponsor:
Clementia Pharmaceuticals Inc.
Conditions:
Fibrodysplasia Ossificans Progressiva
Eligibility:
All Genders
6+ years
Phase:
PHASE2
Brief Summary
Fibrodysplasia ossificans progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formatio...
Detailed Description
The primary objective is to evaluate the ability of different doses of palovarotene to prevent HO at the flare-up site in subjects with FOP as assessed by plain radiographs. This is a Phase 2, multi-...
Eligibility Criteria
Inclusion
- Written, signed, and dated informed subject/parent consent or age-appropriate assent.
- Subjects clinically diagnosed with classic Fibrodysplasia Ossificans Progressiva (FOP).
- Symptomatic onset of a distinct flare-up within 7 days of Study Day 1 (start of study drug) and defined by the presence of at least two of six of the following symptoms: pain, soft tissue swelling, decreased range of motion, stiffness, redness, and warmth. Flare-up must be confirmed by the physician at the Screening visit.
- Flare-up is at an appendicular area (upper or lower extremity), abdomen, or chest; and subject has received, is receiving, or is willing to receive treatment per standard of care, which may or may not include oral prednisone (2 mg/kg PO to a maximum dose of 100 mg daily) for 4 days.
- Abstinent or using two highly effective forms of birth control.
- Subjects must be accessible for treatment and follow-up. Subjects living at distant locations from the investigational site must be able and willing to travel to a site for the initial and all follow-up visits.
Exclusion
- Weight \<20 kg.
- Intercurrent non-healed fracture at any location.
- Complete immobilization of joint at site of flare-up.
- The inability of the subject to undergo imaging assessments using plain radiographs.
- If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or unwilling to discontinue use of these products for the duration of the study.
- Exposure to synthetic oral retinoids in the past 30 days prior to Screening (signature of the informed consent).
- Concurrent treatment with tetracycline due to the potential increased risk of pseudotumor cerebri.
- History of allergy or hypersensitivity to retinoids or lactose.
- Concomitant medications that are inhibitors or inducers of CYP450 3A4 activity.
- Amylase or lipase \>1.5x above the upper limit of normal or with a history of chronic pancreatitis.
- Elevated aspartate aminotransferase or alanine aminotransferase \>2.5x the upper limit of normal.
- Fasting triglycerides \>400 mg/dL with or without therapy.
Key Trial Info
Start Date :
July 14 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 23 2016
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT02190747
Start Date
July 14 2014
End Date
May 23 2016
Last Update
February 16 2021
Active Locations (4)
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1
University of California San Francisco, Division of Endocrinology and Metabolism
San Francisco, California, United States, 94143
2
University of Pennsylvania, Center for Research in FOP & Related Disorders
Philadelphia, Pennsylvania, United States, 19104
3
Hôpital Necker-Enfants Malades, Department of Genetics
Paris, France
4
The Royal National Orthopaedic Hospital, Brockley Hill
Stanmore, Middlesex, United Kingdom, HA7 4LP