Palovarotene

FOP is caused by a mutation in the ACVR1 gene that encodes for the ACVR1/ALK2 receptor. This receptor is part of the bone morphogenetic protein (BMP) signaling pathway and is critical in the regulation of cartilage and bone development and growth. The mutation leads to increased activity of the ACVR1/ALK2 receptor. The mutated, overactive receptor leads to excess phosphorylation of Smads 1/5/8 and enhanced signals to the nucleus to form heterotopic bone. Palovarotene binds to RARγ which decreases levels of Smad 1/5/8 proteins signaling, repressing excess BMP which may prevent the formation of abnormal new bone.

About the Clinical Trial

The Phase 3 trial is a global, multi-center, open-label (no placebo) trial. Approximately 80 adults and children over 4 years of age with the classic FOP mutation (R206H mutation) will be needed to complete the Phase 3 clinical trial. The trial is designed to evaluate whether a daily dosing regimen of palovarotene, with higher doses during times of flare-ups, will reduce the formation of new HO.

Participating Clinical Sites

University of California San Francisco

San Francisco, California, United States, 94143

Contact: Tea Chan

Principal Investigator: Edward Hsiao, MD, PhD

Children's Hospital of Philadelphia 

Philadelphia, Pennsylvania, United States, 19104

Contact: Edna Mancilla, MD

Principal Investigator: Edna Mancilla, MD

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Contact: Katherine S. Toder, CCRC

Principal Investigator: Mona Al Mukaddam, MD, MS

Toronto General Hospital

Toronto, Ontario, Canada, M5G 2C4

Contact: Irene Ho

Principal Investigator: Angela Cheung, MD, PhD

Groupe Hospitalier Necker Enfants Malades 

Paris, France, 75015

Contact: Genevieve Baujat, MD

Principal Investigator: Genevieve Baujat, MD

Hospital Universitari i Politècnic La Fe

Valencia, Avinguda De Fernando Abril Martorell, Nº 106, Spain, 46026

Contact: Inmaculada Calvo, MD, Ph

Principal Investigator: Inmaculada Calvo, MD, PhD

Norrlands Universitetssjukhus 

Umeå, Sweden, SE-90185

Contact: Staffan Berglund, MD, PhD

Principal Investigator: Staffan Berglund, MD, PhD

Royal National Orthopaedic Hospital, Brockely Hill 

Stanmore, United Kingdom, HA7 4LP

Contact: Richard Keen, MD

Contact: Jackie Vinton

Principal Investigator: Richard Keen, MD

Eligibility Criteria Icon
Eligibility Criteria*
  • AGE: 4-60
  • DISEASE ACTIVITY: No flare symptoms in prior 4 weeks
  • MUTATIONS: R206H (non R206H may participate in a sub-study)
Study Design Icon
Study Design*
  • STUDY TYPE:  Interventional
  • RANDOMIZED STUDY:  No
  • PLACEBO CONTROLLED:  No
  • LENGTH OF PARTICIPATION: 24 months
  • NUMBER OF STUDY VISITS: 6
Status Icon
Status

Phase 3, Enrolling

Therapy Approach Icon
Therapy Approach

Smad inhibitor

Study Sponsor Icon
Study Sponsor

Clementia Pharmaceuticals

Q and A icon
Q & A

Q: Who can participate in the palovarotene trial?
A: Answer here

Q: In what countries will the study be conducted?
A: Answer here

Q: If I’m currently enrolled in Clementia’s Natural History Study, can I participate in the palovarotene Phase 3 trial?
A: Individuals participating in Clementia’s Natural History Study who meet the enrollment criteria can enroll in the MOVE study.

Q: If I participated in the palovarotene Phase 2 program, can I enroll in the Phase 3 trial?
A: Answer here

Q: How long will the palovarotene Phase 3 trial last?
A: Answer here

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