Bisphosphonate Therapy for Osteogenesis Imperfecta

Indiana University School of Medicine

Status and phase

Completed

Phase 4

Conditions

Osteoporosis

Paget Disease of Bone

Osteogenesis Imperfecta

Treatments

Drug: Pamidronate

Drug: Alendronate

Study type

Interventional

Funder types

Other

Identifiers

NCT00159419

9902-30

Details and patient eligibility

About

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.

Full 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.

Enrollment

18 patients

Sex

All

Ages

3 to 21 years old

Volunteers

No Healthy Volunteers

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
- Dentinogenesis 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