Ignite Creation Date:
2024-05-05 @ 9:42 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00005901
Status:
COMPLETED
Last Update Posted:
2016-03-01
First Post:
2000-06-06
Brief Title:
Pamidronate to Treat Osteogenesis Imperfecta in Children
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Trial of Pamidronate in Children With Osteogenesis Imperfecta
Status:
COMPLETED
Status Verified Date:
2016-02
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:
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 pamidronate 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
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
Detailed Description:
Osteogenesis imperfecta is an inherited disorder of connective tissue in which affected individuals synthesize abnormal type I collagen This results in the formation of abnormal bone matrix and a predisposition to bony fractures Many unsuccessful attempts have been made to increase the bone mineral density of these individuals in the hope that this will improve the strength of their bones and result in a decreased fracture rate
Bisphosphonates are synthetic analogs of pyrophosphate which have an affinity for hydroxyapatite These drugs act primarily on osteoclasts impairing the development of immature osteoclasts and the function of mature osteoclasts as well as depressing chemical signaling to adjacent cells resulting in a shift in the balance of deposition and resorption in bone Conditions for which bisphosphonates are being used in children include four broad categories 1 a primary defect in bone mineralization 2 bone matrix abnormalities 3 bone abnormalities due to systemic disease or the effects of treatment of systemic disease or 4 significant soft tissue calcification with no bone abnormality
Pamidronate an aminobisphosphonate is a potent inhibitor of bone resorption The purpose of this protocol is to evaluate the effectiveness of pamidronate in children with types III and IV osteogenesis imperfecta when the cycle time is varied We plan to compare children treated with pamidronate on an every-three-month infusion cycle to children treated every six months with the same dose per infusion We also plan to continue to compare children treated with pamidronate and growth hormone to children treated with pamidronate alone
Bisphosphonates are synthetic analogs of pyrophosphate which have an affinity for hydroxyapatite These drugs act primarily on osteoclasts impairing the development of immature osteoclasts and the function of mature osteoclasts as well as depressing chemical signaling to adjacent cells resulting in a shift in the balance of deposition and resorption in bone Conditions for which bisphosphonates are being used in children include four broad categories 1 a primary defect in bone mineralization 2 bone matrix abnormalities 3 bone abnormalities due to systemic disease or the effects of treatment of systemic disease or 4 significant soft tissue calcification with no bone abnormality
Pamidronate an aminobisphosphonate is a potent inhibitor of bone resorption The purpose of this protocol is to evaluate the effectiveness of pamidronate in children with types III and IV osteogenesis imperfecta when the cycle time is varied We plan to compare children treated with pamidronate on an every-three-month infusion cycle to children treated every six months with the same dose per infusion We also plan to continue to compare children treated with pamidronate and growth hormone to children treated with pamidronate alone
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 00-CH-0136 | OTHER | NIH NICHD IRB | None |