Bisphosphonate Therapy for Osteogenesis Imperfecta

Osteoporosis Clinical Trial

Official title:

Bisphosphonate Therapy for Osteogenesis Imperfecta

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00159419
• Other study ID #: 9902-30
• Status: Completed
• Phase: Phase 4
• First received: September 7, 2005
• Last updated: September 26, 2011
• Start date: August 1999
• Est. completion date: August 2008

Study information

• Verified date: September 2011
• Source: Indiana University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." We, the researchers at Indiana University School of Medicine, are characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate therapy in a group of children with OI and also in children with other disorders that result in low bone mass and fractures.

Description:

The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." OI is an inherited disorder of collagen synthesis. Collagen is the major structural protein of the matrix of tendons, skin, and bones. Affected persons have low bone mineral density (and experience multiple fractures and progressive bony deformity). In its most severe form, the disorder is lethal in infancy. We plan to characterize the changes effected by oral bisphosphonate therapy and compare them to a regimen of intravenous bisphosphonate therapy in a group of children with OI.

Additionally, we have begun to treat patients with OI and other conditions of low bone mineralization for age who are not eligible for the standard protocol (too young, history of abdominal pain, etc.) with bisphosphonate. We also plan to screen the parents and siblings of our patients diagnosed with osteogenesis imperfecta, in order to determine if they also have osteoporosis.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: August 2008
• Est. primary completion date: August 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 21 Years

Eligibility

Inclusion Criteria:

• Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) <2.5 standard deviations (SD) for age plus two of the following:

• Family history of OI

• Frequent fractures

• Blue sclerae

• Multiple wormian bones on skull x-ray

• Hearing disturbance

• Dentogenesis imperfecta

• Age between 3 and 21 years at the start of the study period.

• Children must be able to swallow whole tablets

• Parents of children must be able to understand protocol and give informed consent.

Exclusion Criteria:

• Therapy with bisphosphonates during the past 12 months.

• Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.

• Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bone Diseases
• Osteitis Deformans
• Osteogenesis Imperfecta
• Osteoporosis
• Paget Disease of Bone

Intervention

Drug:
• Alendronate
10 mg po q day
• Pamidronate
1mg/kg/day x 3days

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Indiana University School of Medicine

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bone mineral density measured 4 times monthly		6 years	Yes
Primary	fracture rates		6 years	Yes
Secondary	Audiologic effects (annual)		6 years	Yes
Secondary	dental effects (annual)		6 years	Yes
Secondary	pain assessments		6 years	No
Secondary	bone turnover assessments		6 years	Yes