A Randomized Double Blind Placebo Controlled Multi-Center Parallel Group Study of One Year Duration Followed by Two Years of open-label Treatment to Determine the Safety and Efficacy of Orally Administered 2.5 mg or 5.0 mg Daily Risedronate

Institutional Review Board, Hospital for Special Surgery

March 07, 2006

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Principal Investigator

Cathleen L. Raggio, MD

Co-Investigators

Joseph Lane, MD
Helene Pavlov, MD
Peter Bullough, MD
Erin Carter, MS

Summary

The primary objective of this study of patients with Osteogenesis Imperfecta (OI) is to determine the effectiveness of Risedronate, a bisphosphonate, in decreasing fracture rate in children >4 to <16 years of age with Osteogenesis Imperfecta (OI).
Approximately 124 patients are to be enrolled across 15-20 study centers located in North America, Europe, and South America. Patients may be enrolled through August of 2006. After being enrolled, the duration of the study and follow-up is three years.
The secondary objectives include safety and tolerability, laboratory profiles, changes from baseline in bone age, and annualized growth velocity from baseline.

Inclusion/Exclusion Criteria

Inclusion Criteria:

• Children >4 to <16 years old diagnosed with mild OI (type I or type IV);
  
• At least 2 evaluable lumbar spine vertebral bodies (L1-L4 without fracture or degenerative disease);
  
• If female (post-menarche/sexually active), must agree to use a reliable form of contraceptive during the study and have a negative serum/urine pregnancy test at screening;
  
• All subjects and parents/legal guardians must be able and willing to provide a valid written informed consent/assent form.

Major Exclusion Criteria:

• History of Bisphosphonate use within 1 year of enrollment (except single-dose oral bisphosphonates, Risedronate or Alendronate)
  
• Osteoporosis, secondary to diseases other than OI or drug therapies
  
• Clinically-significant abnormal laboratory results or use of certain drugs/medications
  
• Participation in another clinical trial within 3 months of enrollment.

Contact Information

Erin M. Carter, MS
Clinical Coordinator/Genetic Counselor
The Kathryn O. and Alan C. Greenberg Center for Skeletal Dysplasias
212.774.7332 or CSD@hss.edu