BBD Longitudinal Study of Osteogenesis Imperfecta

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorBaylor College of Medicine

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

Individuals who are unable to return for their scheduled follow up visits.

Individuals with skeletal dysplasias other than OI

Individuals with OI and a second genetic or syndromic diagnosis

Use of a bone-acting treatment agent such as bisphosphonates, calcitonin, calcitriol, fluoride, etc., within one year of enrollment.

Trial population

Individuals with a diagnosis of Osteogenesis Imperfecta are eligible to enroll in the natural history study.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

Natural History of OI

The molecular basis of the brittle bone disease will be correlated with phenotype, disease progression and response to current standard of care therapies.

Time frame
10 years

Secondary outcomes

1

Incidence and progression of scoliosis in OI

Incidence and progression of scoliosis in OI analyzed by subtype and Cobb Angle assessment

Time frame
10 years
2

Number Vertebral compression fractures in OI HaploInsufficiency

Number and location of Vertebral compression fractures in OI-HI

Time frame
10 years
3

Incidence of Oral and craniofacial anomalies

Incidence and progression of oral and craniofacial anomalies as captured by panorex and dental exam

Time frame
10 years
4

Satisfaction of Oral Health 15Y+

Satisfaction of Oral health as measure by the OHIP 20

Time frame
10 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Baylor College of Medicine

Lead sponsor

Shriners Hospitals for Children

Collaborator

Hospital for Special Surgery, New York

Collaborator

Children's National Research Institute

Collaborator

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Collaborator

University of California, Los Angeles

Collaborator

Oregon Health and Science University

Collaborator

University of Nebraska

Collaborator

Alfred I. duPont Hospital for Children

Collaborator

University of South Florida

Collaborator

Phoenix Children's Hospital

Collaborator

Marquette University

Collaborator