On June 8, 2022, BioCryst Pharmaceuticals announced that they have received Fast Track Designation from the U.S. Food & Drug Administration (FDA) for BCX9250. Learn more

On May 27, 2022 Regeneron Pharmaceuticals submitted the OPTIMA trial to ClinicalTrials.gov. The trial is a phase III study to examine the safety and efficacy of garetosmab versus placebo administered intravenously (IV) in adult participants with FOP. Learn more 

There are four other FOP clinical trials enrolling FOP patients:

STOPFOP Trial - A Phase 2, 6-month double-blind randomized controlled trial of AZD0530 versus placebo, followed by a 12-month open label extension phase with sites in Germany, The Netherlands and United Kingdom. Learn more

INCB000928 Trial - A Phase 2, 24-week double-blind randomized trial of INCB000928 versus placebo, followed by a 52-week open label extension phase. There are 18 sites listed with 1 actively recruiting. Learn more

Ipsen's FALKON Trial - A Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130. There are 14 sites currently listed with 12 actively recruiting. Learn more 

Ipsen's PIVOINE Trial - A palovarotene Rollover Study open only to those who completed Ipsen's MOVE Trial. There are 10 sites currently listed with 4 actively recruiting. Learn more 

IFOPA Clinical Studies & Trials Resources

You should always discuss your participation in clinical studies and trials with your doctor. The IFOPA has developed these resources to educate you and support your decision-making.

• The Drug Development Journey: How Medicines Become Treatments
• Exploring Clinical Trials for FOP 
  
• Benefit, Risk and Hope in Clinical Trials for FOP presented by Dr. Fred Kaplan, University of Pennsylvania, at the 2021 FOP Family Gathering 
  
• Patient and Family Involvement in the Drug Development and Approval Process presented by Laura Iliescu, Associate Director of Patient Advocacy Strategy, ICON, at the 2021 FOP Family Gathering 
  
• What to Ask When You're Interested in a Clinical Trial - a resource shared by PRA Health Sciences (now ICON) 
  
  

If you have additional questions, please send an email to [email protected].