Investigators Currently Searching for Patients
Enrolling in a study is a personal decision that may have risks or benefits. The FD/MAS Alliance provides this page as a resource for patients and families. Inclusion of a study on this webpage is not an endorsement or recommendation by the FD/MAS Alliance. Researchers with IRB-reviewed or Ethics Committee-approved studies may request to be listed via email to info@fibrousdysplasia.org.
Share Your Experience with Fibrous Dysplasia/McCune-Albright Syndrome
Are you a Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS) patient or caregiver, if Ultragenyx Pharmaceutical invites you to participate in an upcoming virtual advisory board meeting, hosted by Ultragenyx Pharmaceutical. During this virtual meeting, they will discuss the particular challenges faced by and needs of individuals living with FD/MAS, such as the progression of symptoms, pain management or accessing care. Click here for more information.
Harvard Medical School and the National Institutes of Health Seek to Understand the Causes of Pain in FD/MAS Patients
Researchers are trying to recruit study participants for a research study that aims to understand the causes of pain in Fibrous Dysplasia/McCune Albright Syndrome (FD/MAS) patients. Adolescent and adult patients diagnosed with (FD/MAS with or without pain) can participate in the research study. Participants will make up to two visits (approximately 2-3 hours long) to the Boston Children’s Hospital. Compensation, travel accommodations, and reimbursements are available. You can read more about the study here. For more information, please contact the research group at jaymin.upadhyay@childrens.harvard.edu or by phone at 917-736-1541.
FD/MAS Patient Registry Seeks FD patients with and without MAS.
Patients and caregivers can enroll in this IRB-approved study. FD/MAS Patient Registry has 900+ participants who have enrolled. The Registry allows them to fill out surveys and share medical data in a secure repository. Information on this ongoing research project launched by the FDF can be found at www.fdmasregistry.org
Study Contacts:
Tovah Burstein, Study Coordinator and Program Assistant, registry@fibrousdysplasia.org
Carmel Shemmesh-Rafalowsky, Primary Investigator and FD/MAS Alliance Board Member, PI.Registry@fibrousdysplasia.org
Click here for Steering Committee
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
The natural history of this disease is poorly described and there are no clearly-defined systemic therapies for bone disease. The purpose of this study is to define the natural history of the disease with or without treatment.
Study contacts:
Lori C. Guthrie, RN (301) 594-0844 guthriel@mail.nih.gov
Alison M. Boyce, MD (301) 443-2700 alison.boyce@nih.gov
National Institutes of Health
National Institute of Dental and Craniofacial Research (NIDCR)
A Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia
This study is meant to test a drug (burosumab) in people with FD who have low blood phosphate levels. Participants will visit the NIH 3 times in 48 weeks. Each visit will last 5 to 7 days. For more detail visit the study listing here.
Study contacts:
Olivia De Jong (240) 595-2764 olivia.dejong@mail.nih.gov
Study Primary Investigator: Alison M. Boyce, MD (301) 443-2700 alison.boyce@nih.gov
National Institutes of Health
National Institute of Dental and Craniofacial Research (NIDCR)
A Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children with Fibrous Dysplasia
This is a study to test a study drug (denosumab) in children ages 4 to 14 who are diagnosed with FD/MAS. Participants will have a screening visit at the NIH clinic or by telehealth. Their medical history will be reviewed. They will stay overnight in the hospital 4 times in 76 weeks. Each stay will last 5 to 7 nights. Participants will also visit a local lab for blood and urine tests every 4 weeks during the study and will receive denosumab once every 4 weeks for 48 weeks. For more information, visit the study listing.
Study contacts:
Alison M. Boyce, MD (301) 443-2700 alison.boyce@nih.gov
National Institutes of Health
RUDY Study UK Seeks Patients with Rare Bone Diseases
Rudy is a study in rare diseases of the bones, joints, and blood vessels. Headed up by a research team at the University of Oxford, Rudy aims to transform clinical care for participants through patient-driven research.
Study contact:
Kassim Javaid
07775 541615 / 01865 223407
https://research.ndorms.ox.ac.uk/rudy/library
Additional Investigators May Be Seeking Patients
The federal government sponsors the database ClinicalTrials.gov to help people locate scientists studying a wide range of medical conditions and diseases.
If you would like to contribute to the accumulation of scientific knowledge about Fibrous Dysplasia, and McCune Albright Syndrome, which will result in better diagnosis and treatment of people with your condition, you should check it out. Follow the 3 steps below to access the appropriate regions of ClinicalTrials.gov.
- Type the following URL into your browser, or click here: http://clinicaltrials.gov/ct2/search .
- You will see a box below the following statement in the middle of your screen: “Enter a word or phrase, such as the name of a medical condition or intervention.” Type in ” “Fibrous dysplasia,” or “McCune Albright Syndrome.” Take care to spell the search term correctly. Then push the SEARCH button.
- A new web page will open, listing studies that are ongoing and currently recruiting participants as well as those that are completed. Scroll through them and click on ongoing studies to find out further information.
