Status:
COMPLETED
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
Lead Sponsor:
Clementia Pharmaceuticals Inc.
Conditions:
Fibrodysplasia Ossificans Progressiva
Eligibility:
All Genders
4+ years
Phase:
PHASE3
Brief Summary
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelli...
Detailed Description
One of the primary objectives was to evaluate the efficacy of palovarotene in decreasing new HO in participants with FOP as assessed by low-dose, whole body computed tomography (WBCT), excluding head,...
Eligibility Criteria
Inclusion
- Key
- Written, signed, and dated informed subject/parent consent; and for subjects who are minors, age-appropriate assent (performed according to local regulations).
- Males or females at least 4 years of age.
- No flare-up symptoms within the past 4 weeks, including at the time of enrollment.
- Abstinent or using two highly effective forms of birth control.
- Accessible for treatment and follow-up; able to undergo all study procedures including low-dose WBCT (excluding head) without sedation.
- Key
Exclusion
- Weight \<10 kg.
- Concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as imatinib.
- Amylase or lipase \>2x above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
- Elevated aspartate aminotransferase or alanine aminotransferase \>2.5x ULN.
- Fasting triglycerides \>400 mg/dL with or without therapy.
- Female subjects who are breastfeeding.
- Subjects with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
- Simultaneous participation in another clinical research study (other than palovarotene studies) within 4 weeks prior to Screening; or within five half-lives of the investigational agent, whichever is longer.
- Any reason that, in the opinion of the Investigator, would lead to the inability of the subject and/or family to comply with the protocol.
Key Trial Info
Start Date :
November 30 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 7 2022
Estimated Enrollment :
107 Patients enrolled
Trial Details
Trial ID
NCT03312634
Start Date
November 30 2017
End Date
September 7 2022
Last Update
November 29 2023
Active Locations (16)
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1
University of California San Francisco, Division of Endocrinology and Metabolism
San Francisco, California, United States, 94143
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
4
University of Pennsylvania, Internal Medicine
Philadelphia, Pennsylvania, United States, 19104