Clinical Studies and Trials: Your Choice, Your Role, Your Responsibility
Adam Sherman, Director of Research Development & Partnerships, provides an overview of the drug development and approval process and the role that clinical studies and trials play. Learn more about how to select which study or trial is right for you.
Regeneron's LUMINA-1 Trial
Learn more about Regeneron's LUMINA-1 Trial led by the principal investigator, Dr. Bob Pignolo, at the Mayo Clinic LUMINA-1 Trial site. Please note the following update: Slide 21 - Go to their page on ClinicalTrials.gov to find the recruiting status of “A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva (LUMINA-1)."
Clementia's MOVE Trial (NO LONGER RECRUITING PATIENTS)
Learn more about Clementia's MOVE Trial led by the principal investigator, Dr. Ed Hsiao, at the University of California, San Francisco MOVE Trial site. Please note the following corrections: Slide 6 - Total number of flare ups is 42 (not 18); Slide 7 - Total number of flare ups is 140 (not 106); Slide 11 - Sweden is currently enrolling (not The Netherlands).
Hsiao Lab at the University of California, San Francisco
The Hsiao Lab at UCSF has a variety of ongoing clinical studies related to the genetics and disease progression in FOP. Families interested in participating should contact Dr. Edward Hsiao directly at firstname.lastname@example.org to learn more about the research.
The FOP Registry, sponsored by the IFOPA, collects disease information directly from individuals with FOP through a secure website. Data from the Registry will be used to help researchers, clinicians and drug developers to better understand FOP. Learn more.
To read about the IFOPA's support of clinical trials, click here.
Are You Interested in Participating in a Clinical Trial?
After many years of successful basic and translational research, we have begun an important and exciting era of clinical trials for FOP. Clinical trials designed to advance the understanding of FOP and potential treatments are taking place around the world. The IFOPA is dedicated to providing you with information on clinical trials. There are a number of ways you can stay informed:
- Learn more about clinical trials on this website. See our list of active clinical trials for FOP. Check back regularly for updates.
- Participate in the FOP Registry. The registry will give you the option to indicate whether you want to be contacted about research opportunities.
- “Like” the IFOPA Facebook page and watch for announcements
- Visit the www.clinicaltrials.gov website, which is a comprehensive list of clinical studies taking place around the world. Search for FOP clinical trials using the keyword “fibrodysplasia.”
Why Participate in Clinical Trials?
Clinical trials give patients an opportunity to play a more active role in their own health care, gain access to potential treatments before they are widely available, and help others by contributing to medical research. There are also risks to clinical trials. Before enrolling in a clinical trial it is important to understand the potential risks and benefits, and to discuss your options with your doctors and your family.
Please let us know if we at the IFOPA can help you get answers to your questions about a clinical trial so that you can make an informed decision about whether to participate. We are committed to sharing up-to-date information and facilitating dialogue with appropriate experts to enable you to make an informed choice. You can reach us by email at email@example.com or by phone (407) 365-4194.
For general information about clinical trials, please visit the National Institutes of Health site on clinical trials.
The Center for Information and Study on Clinical Research Participation (CISCRP) is an independent non-profit organization dedicated to educating and informing the public and patients about clinical research: www.ciscrp.org/education-center/
FOP Clinical Trials
The most up-to-date place to find information on clinical studies and trials that are active in the United States is www.clinicaltrials.gov. This page lists all active clinical trials for individuals living with FOP.
A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
Interventions: Drug: REGN2477; Drug: Matching Placebo
Investigator-initiated Clinical Trial of NPC-12T for Fibrodsyplasia Ossificans Progressiva
Interventions: Drug: NPC-12T (Rapamycin); Drug: Placebo
The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry
Learn more about the Regneron LUMINA-1 trial, including a list of frequently asked questions.
Active, Not Recruiting
An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Intervention: Drug: Palovarotene; Drug: Placebo
An Open-Label Extension Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Intervention: Drug: Palovarotene dose level 1; Drug: Palovarotene dose level 2; Drug: Palovarotene dose level 3
An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in FOP Subjects in France
Invervention: Drug: Palovarotene dose level 1; Drug: Palovarotene dose level 2
A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
Intervention: Drug: Palovarotene
Urine Sample Collection From FOP Patients