Official Title: “Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children”

Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures.

There are no approved drugs for the treatment of OI in children, even though some intravenous (IV) bisphosphonates are used off-label in some countries. In a single dose, pharmacokinetic study, data showed that risedronate was well tolerated in 28 children with OI. This three year study will test the safety and efficacy of risedronate in the treatment of children with OI. For the first year, patients will be randomized to the risedronate and placebo groups in a 2:1 ratio (two groups will receive risedronate, and one group will receive placebo). For the second and third years of the study, all patients will receive risedronate.

Study Type: Interventional

Study Design: Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study

Study Primary Completion Date: March 2008

Intervention(s) in this Clinical Trial

• Drug: risedronate sodium (Actonel)
  • risedronate tablet once a day for one year followed by risedronate once a day for two years
• Drug: Placebo
  • placebo tablet once a day for one year followed by risedronate once a day for two years

Arms, Groups and Cohorts in this Clinical Trial

• Placebo Comparator: 1
  • placebo tablet, once a day for one year than for two years open label risedronate
• Experimental: 2
  • risedronate tablet, once a day for one year than for two years open label risedronate once a day

Primary Measures

• The primary objective of the study is to determine the efficacy of risedronate compared to placebo in children 4 to <16 years of age with osteogenesis imperfecta as assessed by percent change from Baseline in lumbar spine bone mineral density at Month 12
  • Time Frame: one year
    Safety Issue?: No

Inclusion Criteria:

• OI diagnosis
• increased risk of fracture: either has a history of at least 1 radiographically confirmed, non-traumatic or low impact fracture plus low bone mineral density (BMD) or has very low BMD with or without a history of fractures.

Exclusion Criteria:

• Any bisphosphonate use within one year of enrollment

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 4 Years

Maximum Age for this Clinical Trial: 15 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: Procter and Gamble

Overall Clinical Trial Officials and Contacts

Dietrich H Wenderoth, MD Study Director Procter and Gamble  

Information obtained from ClinicalTrials.gov on August 21, 2008

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00106028

Study ID Number: 2003100

ClinicalTrials.gov Identifier: NCT00106028

Health Authority: United States: Food and Drug Administration