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.
Researchers at Boston Children’s Hospital Seek to Explore FD/MAS and Pain in Adults
Researchers are trying to recruit study participants for a research study that aims to understand the benefit of a biopsychosocial treatment approach for pain in Fibrous Dysplasia/McCune Albright Syndrome (FD/MAS) patients. Adults ages 18 to 50 years diagnosed with FD or MAS, can participate in the research study. Participants will be asked to come to Boston for two day visits at three points during the study. Participants will be asked to come to Boston for as many as three study visits. They will receive travel reimbursement of up to $2,000 per visit and compensation of $350 per visit.
Inclusion criteria (FD/MAS Patients).
(i) 18-50 years of age
(ii) Male or female
(iii) Patients with confirmed diagnosis of FD or MAS via genetic testing, clinical assessment, or imaging
(iv) No treatment with an investigational product
(v) Physician determination that patient is safe to undergo study procedures (Dr. Sethna, Dr. Mannstadt)
(vi) Ability to provide informed consent
Exclusion Criteria (FD/MAS Patients).
(i) Active substance misuse (ETOH, opioids, sedatives, this does not include cannabis), not currently in treatment
(ii) Significant cognitive deficits that would limit ability to engage in therapy
(iii) Severe psychopathology including active suicidal ideation, self-injurious behavior or psychosis, active eating disorder, inability to maintain weight or hydration,
(iv) Certain presentations of functional neurologic disorders that would limit ability to provide effective care, safe treatment including: active seizures, or non-epileptic seizures uncontrolled fainting, episodes of unconsciousness
(v) Presence of craniofacial or upper spine metal implants.
For more information, please contact the research group at jaymin.upadhyay@childrens.
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:
Olivia de Jong, RN (301) 594-0844 olivia.dejong@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.