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Home > "E" Clinical Trials Conditions > Evaluation and Intervention for the Effects of Osteogenesis Imperfecta  Evaluation and Intervention for the Effects of Osteogenesis Imperfecta
Evaluation and Intervention for the Effects of Osteogenesis Imperfecta
For Condition: Dwarfism,Osteogenesis Imperfecta
Status: Recruiting
Sponsor(s): National Institute of Child Health and Human Development (NICHD) ,
Synopsis: Osteogenesis Imperfecta is a genetic disorder occurring in children causing abnormally fragile bones. Patient's with osteogenesis imperfecta often suffer fractures from minimal trauma. These patient's can have deformities of long bones, blueness of the white portion of the eye (sclerae), weak ligaments, and deafness due to diseased bones in the inner ear (otosclerosis). Growth deficiency is a hallmark feature of Osteogenesis Imperfecta. Growth patterns have been defined for some of the different types of Osteogenesis Imperfecta. At about 1 year of age, children with Types III and IV Osteogenesis Imperfecta show a predictable leveling off (plateau) of their growth rate. Children with Type IV Osteogenesis Imperfecta begin to resume normal growth rate at about four to five years, but they will not "catch up" to a normal height. Children with Osteogenesis Imperfecta Type III will remain at a leveled off growth rate. The reason for the leveling off (plateau) of the growth rate is unknown. The purposes of this study will concentrate on four areas of research; genetics, rehabilitation, growth, and the natural history of the multiple secondary features of Osteogenesis Imperfecta. This study will be broken up into 2 steps or phases. Phase I will focus on children from birth to age 5, and Phase II will concentrate on children ages 5 - 18 years. One of the objectives in Phase I will be to determine the effects of long leg bracing in children with osteogenesis imperfecta. The study will observe the patient's ability to do activities while standing upright as well as the development of manual skills. In addition the study will observe the alignment of the patient's bones and the bone strength and density.
Details: We propose a two phase longitudinal study for children affected with Sillence Types III and IV OI. Phase I will target children from Birth - 5 years of age, and Phase II is directed toward children ages 5 - 18 years. One objective of Phase I includes a prospective randomized bracing trial to determine the effect of long leg bracing on (1) the timing and progression of upright activity and development of gross motor skills, (2) bony alignment, and (3) bone density. The protocol includes a complete evaluation of the growth plateau experienced by all Types III and IV OI children between the ages of 12 months and 4-6 years. Our long term goal related to this evaluation is to develop a treatment to eliminate the growth plateau. A consistent objective throughout both phases of this study is to obtain a comprehensive assessment of the natural history and progression of the multiple secondary features of this disorder. In addition to radiographic, bone density, physical rehabilitation and dental manifestations, we will assess the neurological, cardiovascular, pulmonary, and audiologic systems. Our objectives to determine the incidence of basilar invagination and to develop a monitoring and management plan for this neurological feature are particularly important. Another consistent objective which spans both phases of this study is the genetic and molecular biology aspect of OI. Patients and their parents will have skin biopsies for collagen studies at the biochemical and molecular level. These studies will provide further information on genotype/phenotype correlation and other variables in OI genetics. As appropriate, bone chips from emergency or elective surgical procedures on the OI children will be used to study osteoblast function in OI.
Eligibility:
Study Type: Observational, Natural History
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: Children will be recruited from the entire United States. Children enrolled in this study will be limited to those with Sillence Types III and IV OI, as determined by clinical and genetic criteria. New patients must be under the age of 2 years to be enrolled and should be expected to attain at least assisted ambulation. EXCLUSION CRITERIA: Children who have had a skin biopsy done for collagen analysis at another facility will be excluded from participation in this study. Children who are clinically too severe to benefit from this program are defined by the following: (a) the ratio of head circumference age (the age for which the child's head or body size falls at the 50th percentile) to body length age is 7:1 or greater. (b) Children who are 24 months of age, and who are unable to sit unsupported for 60 seconds and are unable to demonstrate the ability to prop themselves on upper extremities in the prone position. Once enrolled, parents will be required to agree to postpone all elective orthopedic surgery with the following exceptions: (1) the child has had two fractures of the same lower long bone within 18 months, (2) the bowing angle has increased to 50 degrees or more and bracing is not possible over the curved bone, or (3) the child has a substantial valgus/varus angulation in excess of 30 degrees. Parents will also be required to agree that no orthopedic surgery will be performed without prior discussion with the principal investigator, and members of the NIH OI team. Surgical interventions outside these boundaries will constitute exclusion criteria and the child will be dropped from the study. Failure to comply with these conditions will constitute exclusion criteria.
Total Enrollment: 116
Location and Contact Information:
National Institute of Child Health and Human Development (NICHD) *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Patient and Public Liaison Office 1-800-411-1222
Additional Information:
Study ID Numbers: 970064; 97-CH-0064
Study Start Date: January 13, 1997
Record last reviewed: December 12, 2002
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00001594
Other Osteogenesis Imperfecta Studies:
1. Evaluation and Intervention for the Effects of Osteogenesis Imperfecta
2. Growth Hormone Therapy in Osteogenesis Imperfecta
3. Bisphosphonate Treatment of Osteogenesis Imperfecta
4. Pamidronate to Treat Osteogenesis Imperfecta in Children
Related Studies:
Other Osteogenesis Imperfecta Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Evaluation and Intervention for the Effects of Osteogenesis Imperfecta
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