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Retinoic Acid Receptor Agonist

Palovarotene dose level 2 for Stone Man Syndrome

Phase 2
Waitlist Available
Research Sponsored by Clementia Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up study months 6 and 12 (follow-up component); flare-up weeks 2, 4, 6, 8, 9, and 12 (flare-up component)
Awards & highlights

Study Summary

This trial will test if different doses of a drug called palovarotene can prevent new bone growth in people with Fibrodysplasia Ossificans Progressiva (FOP), a rare, severely disabling disease.

Eligible Conditions
  • Stone Man Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~study months 6 and 12 (follow-up component); flare-up weeks 2, 4, 6, 8, 9, and 12 (flare-up component)
This trial's timeline: 3 weeks for screening, Varies for treatment, and study months 6 and 12 (follow-up component); flare-up weeks 2, 4, 6, 8, 9, and 12 (flare-up component) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part A and Part B - Proportion of flare-up with no new HO ("responders")
Part C - Annualized change in new HO volume
Secondary outcome measures
Duration of active, symptomatic flare-up (start date and end date)
Part A - Change from baseline in amount (area) of new heterotopic bone formed
Bone and Bones
+17 more

Trial Design

4Treatment groups
Experimental Treatment
Group I: Palovarotene dose level 4Experimental Treatment1 Intervention
All participants will receive 5 mg palovarotene for up to 48 months and 20 mg palovarotene for 28 days, followed by 10 mg for 56 days for eligible flare-ups (Part C). Skeletally immature participants will receive weight-adjusted doses.
Group II: Palovarotene dose level 3Experimental Treatment1 Intervention
Participants with less than 90% skeletal maturity received weight-adjusted doses of 20 mg palovarotene for 28 days, followed by 10 mg for 56 days for eligible flare-ups (Part B).
Group III: Palovarotene dose level 2Experimental Treatment1 Intervention
Participants with at least 90% skeletal maturity received 5 mg palovarotene for up to 24 months and 20 mg palovarotene for 28 days, followed by 10 mg for 56 days for eligible flare-ups (Part B).
Group IV: Palovarotene dose level 1 (completed)Experimental Treatment1 Intervention
Participants received 10 mg palovarotene for 14 days, followed by 5 mg palovarotene for 28 days (or weight-based equivalent) for eligible flare-ups (Part A).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Palovarotene dose level 1
2014
Completed Phase 2
~60
Palovarotene dose level 2
2014
Completed Phase 2
~60
Palovarotene dose level 3
2014
Completed Phase 2
~60
Palovarotene dose level 4
2014
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

Clementia Pharmaceuticals Inc.Lead Sponsor
10 Previous Clinical Trials
681 Total Patients Enrolled
Ipsen Medical DirectorStudy DirectorIpsen
257 Previous Clinical Trials
55,163 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
Nature MedicineJournal
Potent Inhibition of Heterotopic Ossification by Nuclear Retinoic Acid Receptor-γ Agonists
Shimono, Kengo, Wei-en Tung, Christine Macolino, Amber Hsu-Tsai Chi, Johanna H Didizian, Christina Mundy, Roshantha A Chandraratna, et al.. 2011. “Potent Inhibition of Heterotopic Ossification by Nuclear Retinoic Acid Receptor-γ Agonists”. Nature Medicine. Springer Science and Business Media LLC. doi:10.1038/nm.2334.
clinicaltrials.govStudy
An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)
2014. "An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02279095.
~5 spots leftby Apr 2025
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