Clinical trials: McCune Albright Syndrome
Fibrous Dysplasia
Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
Background: Fibrous dysplasia (FD) is a disease that affects the bones. It causes bone lesions that can become weak and lead to fractures, deformity, and nerve injuries.
- Trial name
- NCT05419050
- Testing
- denosumab
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Precocious Puberty, Gonadotropin-dependent
(Z)-Endoxifen in Girls With MAS-Associated Peripheral Precocious Puberty
The goal of this clinical trial is to evaluate the safety, tolerability, and blood levels of (Z)-endoxifen in girls with McCune-Albright Syndrome that have peripheral precocious puberty.
- Trial name
- NCT07869732
- Testing
- (Z)-endoxifen
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Atossa Therapeutics, Inc.
Fibrous Dysplasia, Fibrous Dysplasia of Bone
Fibrous Dysplasia: An Epidemiological and Correlational Evaluation of Multimodal Data
Fibrous dysplasia is a benign, pseudotumoral, genetic but non-hereditary condition characterized by the presence of one or more areas of abnormal bone development in which the normal structure is replaced by fibrous tissue.
- Conditions
- Fibrous Dysplasia, Fibrous Dysplasia of Bone, Fibrous Dysplasia/McCune-Albright Syndrome, Mazabraud Syndrome
- Trial name
- FIBR DYSPLASIA (NCT07569731)
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Istituto Ortopedico Rizzoli
Fibrous Dysplasia/McCune-Albright Syndrome
Assessment of Femoral Failure Load and Fracture Risk in Rare Bone Disorders Using MEKANOS Tool. Case Study of Fibrous Dysplasia of Bone/McCune Albright Syndrome
The issue of rare diseases has become a public health priority. In France, a national plan for rare diseases was published in 2004.
- Trial name
- MEKADYS (NCT07507942)
- Testing
- Application of MEKANOS tool to CT scans
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hospices Civils de Lyon
Fibrous Dysplasia, McCune Albright Syndrome
Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry
The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients and families to help answer some of the biggest questions about FD/MAS by completing questionnaires about their lives with FD or MAS.
- Conditions
- Fibrous Dysplasia, McCune Albright Syndrome, Mazabraud Syndrome
- Trial name
- NCT03231644
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Tovah Burstein
McCune-Albright Syndrome
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous tissue or mechanically abnormal bone.
- Trial name
- NCT00001727
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
McCune Albright Syndrome, Polyostotic Fibrous Dysplasia
A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
This study will examine the effect of pegvisomant on growth hormone excess in patients with McCune-Albright syndrome (MAS). Patients with this disease have polyostotic fibrous dysplasia-a condition in which areas of normal bone…
- Trial name
- NCT00017927
- Testing
- Pegvisomant
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Nervous System Disease
PET Imaging of Phosphodiesterase-4 (PDE4) in Brain and Peripheral Organs of McCune-Albright Syndrome
Background: McCune-Albright Syndrome (MAS) is a disorder that affects the bones, skin, and some hormone-producing tissues. It is associated with a mutation in a gene. This gene affects enzymes in the brain and body.
- Trial name
- NCT02743377
- Testing
- Brain PET Imaging with 11C Rolipram, Whole Body PET Baseline with 11C Rolipram, Whole Body PET Blocked with Roflumilast
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- National Institute of Mental Health (NIMH)
Polyostotic Fibrous Dysplasia, Precocious Puberty
Testolactone for the Treatment of Girls With LHRH Resistant Precocious Puberty
The normal changes of puberty, such as breast enlargement, pubic hair and menstrual periods, usually begin between the ages of 9 and 15 in response to hormones produced in the body.
- Trial name
- NCT00001181
- Testing
- Testolactone
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Fibrous Dysplasia/McCune-Albright Syndrome
Pain in Fibrous Dysplasia
Pain remains a common and frequently debilitating symptom, particularly during adulthood of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS).
- Trial name
- NCT04125862
- Testing
- MRI-based Neuroimaging, Non-contrast MRI, 18F-FDG-PET/CT
- Where
- United States
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Polyostotic Fibrous Dysplasia
Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
This study will evaluate the effectiveness of a new bone injection technique for treating bone disease in patients with polyostotic fibrous dysplasia or McCune-Albright syndrome.
- Trial name
- NCT00001851
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Fibrous Dysplasia of Bone
Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone.
- Trial name
- PROFIDYS (NCT00445575)
- Testing
- risedronate
- Where
- 8 sites in Belgium, France and 2 more
- Volunteers
- 80 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Fibrous Dysplasia, McCune Albright Syndrome
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease, consisting of the replacement of normal bone tissue with fibrous tissue.
- Trial name
- DeFiD (NCT05966064)
- Testing
- Denosumab 120 Mg/1.7 Ml Inj
- Where
- Netherlands
- Volunteers
- 82 people
- Sponsor
- Natasha Appelman-Dijkstra
Fibrous Dysplasia of Bone, McCune Albright Syndrome
PAINDYS_Characterizing Pain in Fibrous Dysplasia of Bone/McCune-Albright Syndrome: an Exploratory Pilot Study
Fibrous dysplasia of bone (FD) / McCune-Albright syndrome (MAS) is a rare congenital bone disorder affecting one or multiple bones, caused by a mosaic somatic mutation of the GNAS gene.
- Trial name
- PAINDYS (NCT07476768)
- Testing
- Visit 1 at the center
- Where
- France
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
McCune Albright Syndrome, Polyostotic Fibrous Dysplasia
Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
This study will test the safety and effectiveness of letrozole in treating precocious (early) puberty in girls with McCune-Albright syndrome (MAS).
- Conditions
- McCune Albright Syndrome, Polyostotic Fibrous Dysplasia, Precocious Puberty
- Trial name
- NCT00006174
- Testing
- Letrozole
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Polyostotic Fibrous Dysplasia
Alendronate to Treat Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
This study will evaluate the effectiveness of alendronate in treating the bone abnormality in polyostotic fibrous dysplasia and McCune-Albright syndrome.
- Trial name
- NCT00001728
- Testing
- Fosamax (Alendronate)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
McCune-Albright Syndrome
Histamine Responsiveness in McCune-Albright Syndrome
McCune-Albright syndrome (MAS) is a syndrome caused by a genetic mutation that causes a specific protein in the body called a G protein to be constantly active.
- Trial name
- NCT00318097
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Children's Mercy Hospital Kansas City
Fibrous Dysplasia of Bone, McCune Albright Syndrome
Effectiveness of Medical Management of Fibrous Dysplasia of Bone.
The objective of our study was to assess the effectiveness of our reference center since its constitution. In a retrospective cohort study, we compared the activity of our center, including the time elapsed between diagnosis and…
- Trial name
- Efficience (NCT05422833)
- Testing
- Evaluation and analysis of clinical data
- Where
- France
- Volunteers
- 528 people
- Sponsor
- Hospices Civils de Lyon
Polyostotic Fibrous Dysplasia
Studies on Abnormal Bone From Patients With Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
This study will investigate how a gene mutation (change in DNA) causes the abnormal bone in fibrous dysplasia-a condition in which areas of normal bone are replaced with a fibrous growth similar to a scar.
- Trial name
- NCT00001973
- Where
- United States
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Fibrous Dysplasia Of Bone
Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia
Background: Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems.
- Trial name
- NCT05509595
- Testing
- Burosumab
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)