Ignite Creation Date:
2024-05-06 @ 4:02 AM
Last Modification Date:
2024-10-26 @ 11:42 AM
Study NCT ID:
NCT02432625
Status:
RECRUITING
Last Update Posted:
2023-11-13
First Post:
2015-04-29
Brief Title:
BBD Longitudinal Study of Osteogenesis Imperfecta
Sponsor:
Baylor College of Medicine
Organization:
Baylor College of Medicine
Study Overview
Official Title:
Rare Diseases Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta
Status:
RECRUITING
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
The purpose of this natural history study is to perform a long-term follow-up of a large group of people with osteogenesis imperfecta OI The research aims are
1 To collect natural history data on all individuals enrolled in this longitudinal study The cause of the brittle bone disease will be compared with things like severity various features and response to treatments
2 To determine how often people with type I OI have vertebral compression fractures of the spine
3 To determine how often people with OI develop scoliosis curvature of the spine
4 To determine how often people with OI have problems with teeth alignment and how dental health impacts a persons quality of life
5 To determine the effect of pregnancy in women with OI
There will be a total of 1000 people with OI in this study Participants will be asked to come in every year if 17Y and younger or every other year if 18Y and older for a total of five years
The following information will be collected at the study visits
Birth History and past surgical history Current medical history Scoliosis evaluation Walking ability Questionnaire Dental Quality of Life Questionnaire Scoliosis and fractures Quality of Life Questionnaires Physical development evaluation Medications Use
The following tests will be performed
Physical exam dental exam lung function test hearing test mobility test
The following X-rays will be taken
DEXA scan X-ray of the spine X-ray of the jaw
Biospecimen urine and blood samples will be collected
1 To collect natural history data on all individuals enrolled in this longitudinal study The cause of the brittle bone disease will be compared with things like severity various features and response to treatments
2 To determine how often people with type I OI have vertebral compression fractures of the spine
3 To determine how often people with OI develop scoliosis curvature of the spine
4 To determine how often people with OI have problems with teeth alignment and how dental health impacts a persons quality of life
5 To determine the effect of pregnancy in women with OI
There will be a total of 1000 people with OI in this study Participants will be asked to come in every year if 17Y and younger or every other year if 18Y and older for a total of five years
The following information will be collected at the study visits
Birth History and past surgical history Current medical history Scoliosis evaluation Walking ability Questionnaire Dental Quality of Life Questionnaire Scoliosis and fractures Quality of Life Questionnaires Physical development evaluation Medications Use
The following tests will be performed
Physical exam dental exam lung function test hearing test mobility test
The following X-rays will be taken
DEXA scan X-ray of the spine X-ray of the jaw
Biospecimen urine and blood samples will be collected
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None