Bisphosphonate Therapy for Osteogenesis Imperfecta - Full Text View

Purpose

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.

Condition	Intervention	Phase
Osteogenesis Imperfecta Osteoporosis Paget Disease of Bone	Drug: Alendronate Drug: Pamidronate	Phase 4


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Bisphosphonate Therapy for Osteogenesis Imperfecta

Resource links provided by NLM:

Genetics Home Reference related topics: Paget disease of bone geleophysic dysplasia inclusion body myopathy with early-onset Paget disease and frontotemporal dementia juvenile Paget disease mucopolysaccharidosis type IV osteogenesis imperfecta

MedlinePlus related topics: Osteogenesis Imperfecta Osteoporosis

Drug Information available for: Alendronate sodium

Genetic and Rare Diseases Information Center resources: Osteogenesis Imperfecta Mucopolysaccharidosis Type IV

U.S. FDA Resources

Further study details as provided by Indiana University:

Primary Outcome Measures:

Bone Mineral Density [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
By DXA

Secondary Outcome Measures:

Pain Assessments [ Time Frame: 6 years ] [ Designated as safety issue: No ]
Bone Turnover Assessments [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]


Enrollment:	18
Study Start Date:	August 1999
Study Completion Date:	August 2008
Primary Completion Date:	August 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Alendronate 1 mg/kg po qd rounded to nearest 10 or 20 mg dose	Drug: Alendronate Other Name: fosamax
Active Comparator: Pamidronate 3 mg/kg IV q4 months	Drug: Pamidronate

Detailed 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.

Eligibility


Ages Eligible for Study:	3 Years to 21 Years (Child, Adult)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00159419

Sponsors and Collaborators

Indiana University School of Medicine

Investigators


Principal Investigator:	Linda A DiMeglio, MD, MPH	Indiana University School of Medicine

More Information


Responsible Party:	Linda DiMeglio, MD, MD, Indiana University
ClinicalTrials.gov Identifier:	NCT00159419 History of Changes
Other Study ID Numbers:	9902-30
Study First Received:	September 7, 2005
Results First Received:	November 12, 2013
Last Updated:	February 29, 2016
Health Authority:	United States: Institutional Review Board

Keywords provided by Indiana University:


Osteogenesis Imperfecta Fractures Pediatric Osteoporosis Juvenile Pagets

Additional relevant MeSH terms:


Osteoporosis Osteogenesis Imperfecta Osteitis Deformans Bone Diseases Bone Diseases, Metabolic Musculoskeletal Diseases Osteochondrodysplasias Bone Diseases, Developmental	Genetic Diseases, Inborn Collagen Diseases Connective Tissue Diseases Alendronate Diphosphonates Bone Density Conservation Agents Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 30, 2016