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Protocol Number:
00-CH-0136
- Title:
A Trial of Pamidronate in Children with Osteogenesis Imperfecta
- Number:
00-CH-0136
- Summary:
This study will evaluate the effect of pamidronate-a drug that decreases bone resorption (breakdown)-on osteogenesis imperfecta. This is a genetic disorder of collagen, the major protein in bone. The abnormal collagen causes weak bones, and children with severe osteogenesis imperfecta sustain many fractures throughout their lives. They also have growth deficiency, curvature of the spine, crumbling teeth, hearing loss, easy bruising and heart and lung problems. The study will compare bone density, quality and strength, final adult height, trunk height, and functional ability in children who receive 1) pamidronate every 3 months, 2) tpamidronate every 3 months + growth hormone injections, 3) pamidronate every 6 months, or 4) pamidronate every 6 months + growth hormone injections.
Children 2 years of age and older with severe osteogenesis imperfecta (types III and IV) may be eligible for this study. Those enrolled will be randomly assigned to groups according to age; children two to four years of age will be randomly assigned to receive pamidronate every 3 or every 6 months. Children four years of age and older may participate in the growth hormone treatment groups. These children will continue on growth hormone until they reach their adult height or fail to grow as much as would be expected for someone on growth hormone.
Patients will be followed in the clinic every 3 months for a history, physical examination, X-rays, blood tests, and measurements (weight, head circumference, and bone lengths). Children will receive a 3 to 4 hour infusion of pamidronate through an intravenous catheter (thin flexible tube placed in a vein) once a day for 3 days each visit. (Once inserted, the catheter is left in place to avoid multiple needle sticks for administering the drug and collecting blood samples.) Children who are taking growth hormone will be given the drug at the first treatment visit. At that time, the accompanying parent will be instructed on how to mix the drug and give injections. The child receives an injection 6 days a week (Sunday off).
- Sponsoring Institute:
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National Institute of Child Health and Human Development (NICHD)
- Recruitment Detail
- Type:
Active Accrual Of New Subjects
- Gender:
Male & Female
- Referral Letter Required:
No
- Population Exclusion(s):
None
- Eligibility Criteria:
INCLUSION CRITERIA:
Children enrolled in this study will be limited to those with Sillence types III and IV OI, as determined by clinical and genetic criteria.
Most of the children who will be included in this study are already enrolled in the protocols Evaluation and Intervention for Ambulation, Growth, and Basilar invagination in Osteogenesis Imperfecta (97-CH-0064) and Growth Hormone Therapy in Osteogenesis Imperfecta (92-CH-0034).
Screening of candidates will be based on telephone interviews with a parent, and referral records to include: AP and lateral radiographic films of the lower extremities and spine, family, developmental, fracture and physical history. An NIH clinical screening evaluation will be performed for those children who appear to be appropriate candidates. Patients admitted for this screeing visit who are less than four years of age as well as those older than 4 years of age but not meeting the criteria for inclusion in the growth hormone protocol, protocol 92-CH-0034, will be admitted under protocol 97-CH-0064 (Evaluation and Intervention for Ambulation, growth and basilar Invagination in OI), those older than four years who meet the criteria will be admitted under protocol 92-CH-0034 and will meet the criteria for inclusion into that study.
The inclusion criteria for protocol 92-CH-0034 are as follows: patients must have a clinical/biochemical diagnosis of osteogenesis imperfecta types III or IV, height less than third percentile for age, and radiological evidence that long bone epiphyses have not yet fused.
Patients are excluded from protocol 92-CH-0034 if they have scoliosis of greater than 40% unless scoliosis has been stable over the past two years, or evidence of severe basilar invagination.
Patients with previous exposure to bisphosphonates in outside trials will be considered for participation in this trial.
EXCLUSION CRITERIA:
Inability to comploy with the visit schedule, maintenance of the physical therapy program, and ability to administer and comply with GH injections are central to our analysis of the outcomes of this study. Failure to comply with these conditions will constitute exclusion criteria.
Pregnancy.
- Special Instructions:
Currently Not Provided
- Keywords:
-
Bisphosphonates
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Growth Hormone
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Bone Density
- Recruitment Keywords:
-
Osteogenesis Imperfecta
- Conditions:
-
Osteogenesis Imperfecta
- Investigational Drug(s):
-
Pamidronate (Aredia)
- Investigational Device(s):
- None
- Contacts:
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Patient Recruitment and Public Liaison Office
Building 61 10 Cloister Court Bethesda, Maryland 20892-4754 Toll Free: 1-800-411-1222 TTY: 301-594-9774 (local),1-866-411-1010 (toll free) Fax: 301-480-9793 Electronic Mail:prpl@mail.cc.nih.gov
- Citations:
Not Provided
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Warren Grant Magnuson Clinical Center (CC) National Institutes of Health (NIH)
Bethesda, Maryland 20892. Last update: 10/23/2004
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