Rare Diseases Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta

Status: Recruiting
Location: See all (12) locations...
Study Type: Observational
SUMMARY

Osteogenesis Imperfecta (OI) is a rare disorder of increased bone fragility characterized by fractures with minimal or absent trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. It is seen in both genders and all races. The clinical features of OI represent a continuum varying from perinatal lethality to individuals with severe skeletal deformities, mobility impairments, and very short stature to nearly asymptomatic individuals with a mild predisposition to fractures, normal stature, and normal lifespan. Fractures can occur in any bone, but are most common in the extremities. These disorders can be devastating and progressive and result in deformity, chronic pain, impaired function and loss of quality of life. The overall goal of this study is to answer specific question about the natural history of brittle bone diseases as defined by molecular etiology and to develop the foundation for prospective clinical studies.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Individuals with OI diagnosed by molecular (DNA) analysis OR

• Individuals whose clinical history and radiographs are highly suggestive of OI, but whose diagnosis has not been verified by biochemical or molecular studies

• All study participants between the ages of 3 to 17 years OR

• Study participants 18 years and older with scoliosis

⁃ Dental and Craniofacial Abnormalities in OI component:

⁃ Pregnancy in OI component:

⁃ • Females of reproductive age with mutations in any known gene causing OI, who are contemplating pregnancy within 5 years of enrollment in the Natural History Study OR Females who are pregnant with available pre-pregnancy BMD (within 5 years prior to the first pregnancy visit).

Locations
United States
Arizona
Phoenix Children's Hospital
RECRUITING
Phoenix
California
University of California Los Angeles
RECRUITING
Los Angeles
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Delaware
AI Dupont Hospital for Children
RECRUITING
Wilmington
Florida
University of South Florida
RECRUITING
Tampa
Maryland
Kennedy Krieger Institute / Hugo W. Moser Research Institute
RECRUITING
Baltimore
Nebraska
University of Nebraska Medical Center
RECRUITING
Omaha
New York
Hospital for Special Surgery
RECRUITING
New York
Oregon
Oregon Health and Science University
RECRUITING
Portland
Texas
Baylor College of Medicine
RECRUITING
Houston
Wisconsin
Shriners Hospital for Children, Chicago / Marquette University
RECRUITING
Milwaukee
Other Locations
Canada
Shriners Hospital for Children
RECRUITING
Montreal
Contact Information
Primary
Dianne Nguyen
diannen@bcm.edu
713.798.6694
Time Frame
Start Date: 2015-06-01
Estimated Completion Date: 2031-12-31
Participants
Target number of participants: 1000
Sponsors
Collaborators: University of Nebraska, Children's National Research Institute, Marquette University, Phoenix Children's Hospital, Hospital for Special Surgery, New York, University of South Florida, Oregon Health and Science University, Alfred I. duPont Hospital for Children, Hugo W. Moser Research Institute at Kennedy Krieger, Inc., Shriners Hospitals for Children, University of California, Los Angeles
Leads: Baylor College of Medicine

This content was sourced from clinicaltrials.gov