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Sponsored by: |
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00001305 |
Growth deficiency is a key feature of severe Osteogenesis Imperfecta (OI) and a frequent feature of mild to moderate forms of the disease. The reason that children with OI are short is not fully understood. We do know that details such as the number of fractures suffered or the type of OI do not fully explain the short stature of OI. Growth patterns have been defined for children with OI Types I, III, and IV. At about 12 months of age, children with Types III and IV OI demonstrate a predictable plateau of their linear growth rate. Type IV OI children begin to resume a normal growth rate at about age four to five years, but they will not "catch up" to a normal height, as they have "lost" a significant period of growth. The plateau usually continues for children with Type III OI. The reason for this growth plateau is unknown. There have been no studies which evaluate the growth of OI children in this age range. Our previous studies of growth in OI children have begun at age 5 years.
We have studied growth in OI children for the past 10 years. Different medications have been tried to both stimulate growth and improve bone density. Some children have responded to growth hormone (their growth rate increased by at least 50%) and some did not. The majority of children who did respond were Type IV. However, we need to carefully treat and study more children to try to determine which children will benefit from growth hormone medication.
The Goals of this Study Are:
Median Subject Age (on p. 1 of webpage): 1-15 years (replaces 0-20)
Condition | Intervention | Phase |
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Dwarfism Osteogenesis Imperfecta |
Drug: Humatrope Drug: GRH Drug: Nutropin |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Single Group Assignment, Efficacy Study |
Official Title: | Studies of Growth Deficiency and Growth Hormone Treatment in Children With Osteogenesis Imperfecta Types III and IV |
Estimated Enrollment: | 60 |
Study Start Date: | November 1991 |
Ages Eligible for Study: | 1 Year to 15 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Patients will be recruited with the goal of including at least 10 each of individuals with clinical/biochemical criteria of types III and IV OI who are between 1 and 8 years of age.
Height: Individuals with type III OI have severe short stature by definition; individuals with type IV OI recruited to the study will have height less than the 3rd percentile for age. All individuals will be required to furnish growth records, especially height and head circumference, from at least the preceding two years.
Long bone status: Participants must have radiographic evidence that long bone epiphyses have not yet fused. In addition, 60 degrees or greater angulation of a femur will exclude a child, pending surgical management or medical clearance.
Spine: Prospective participants will be evaluated for scoliosis and spinal compressions. Participants with scoliosis greater than 40 degrees will be excluded unless evidence is presented that the scoliosis has been stable for the prior two years. Participants with corrective rods in their spine will be excluded.
Neuro status: All patients will be co-enrolled in 97-CH-0064, and will be screened for Basilar Invagination through that protocol. Children who are initially screened by spiral CT scan with MRI confirmation and determined to have severe BI will be excluded from participation in this study. Severe BI is defined by NIH data as distortion of the angle between the pons and medulla and or compression of posterior fossa contents. We are only beginning to define the parameters of BI in this population, and we do not know why some children with BI progress in severity and some do not. Until those questions are answered, we feel it would not be prudent to stimulate growth in a child we know to have a severe form of BI at enrollment.
Pulmonary status: All children will be co-enrolled in 97-CH-0064, and will have pulmonary function testing and polysomnograms through that protocol. Tests will be scheduled as required for that protocol; namely, PFTs every 2 years if normal, every year if abnormal, and polysomnograms every 4 years if normal, and every 2 years if abnormal.
Potential participants who have not participated in the growth plateau study will still be eligible for participation in the growth hormone treatment trial. These patients, if entering from outside the protocol, must be between age 4 and 8 years, and must have documented growth records that demonstrate that they have emerged from the growth plateau. The first year in the protocol for these patients will be the pretreatment year, in which they will not receive growth hormone but will come to NIH on the schedule for the pretreatment visits.
EXCLUSION CRITERIA:
Patients who develop scoliosis greater than 40 and/or patients who progress to severe basilar invagination during the study will be removed from the study.
Failure to comply with the outlined procedures (blood draws, endocrine testing, bone biopsies, and visit schedule) is also a criterion for withdrawal from the protocol.
Contact: Patient Recruitment and Public Liaison Office | (800) 411-1222 | prpl@mail.cc.nih.gov |
Contact: TTY | 1-866-411-1010 |
United States, Maryland | |
National Institutes of Health Clinical Center, 9000 Rockville Pike | Recruiting |
Bethesda, Maryland, United States, 20892 |
Responsible Party: | National Institutes of Health ( Joan C. Marini, M.D./National Institute of Child Health and Human Development ) |
Study ID Numbers: | 920034, 92-CH-0034 |
Study First Received: | November 3, 1999 |
Last Updated: | November 8, 2008 |
ClinicalTrials.gov Identifier: | NCT00001305 |
Health Authority: | United States: Federal Government |
Heparin Lock Bone Biopsy Children Collaborative Study |
Osteogenesis Imperfecta Growth Hormone Short Stature Bone Density |
Dwarfism Osteogenesis Imperfecta Osteogenesis imperfecta Collagen Diseases Osteochondrodysplasias Endocrine System Diseases Bone Diseases |
Calcium heparin Musculoskeletal Diseases Genetic Diseases, Inborn Bone Diseases, Developmental Connective Tissue Diseases Endocrinopathy Heparin |