Phase 3
N=221
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
Fibrodysplasia Ossificans Progressiva
Bottom Line
View on ClinicalTrials.gov: NCT03312634 ↗Enrolled (actual)
221
Serious AEs
17.5%
Results posted
Mar 2023
Primary outcome: Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Palovarotene (Drug)
- Age
- Pediatric, Adult, Older Adult · 4+ yrs
- Sex
- All
- Sponsor
- Clementia Pharmaceuticals Inc.
- Primary completion
- Jan 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized New Heterotopic Ossification (HO) |
9427.1; 23720.2 | — |
| SECONDARY Percentage of Participants With Any New HO |
83.5 | — |
| SECONDARY Number of Body Regions With New HO |
3.0 | — |
| SECONDARY Percentage of Participants With Flare-Ups |
64.6 | — |
| SECONDARY Ratio of Flare-Up Per Participant-Month of Exposure |
0.2 | — |
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 swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability.
Eligibility Criteria
Key Inclusion Criteria
- 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 Criteria
- Weight 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.
Data sourced from ClinicalTrials.gov (NCT03312634). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.