Bisphosphonate Therapy for Osteogenesis Imperfecta

Study Description

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Brief 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.

Condition or disease	Intervention/treatment	Phase
Osteogenesis Imperfecta; Osteoporosis; Paget Disease of Bone	Drug: Alendronate; Drug: Pamidronate	Phase 4

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	18 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Bisphosphonate Therapy for Osteogenesis Imperfecta
Study Start Date :	August 1999
Actual Primary Completion Date :	August 2008
Actual Study Completion Date :	August 2008

Arms and Interventions

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm	Intervention/treatment
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

Outcome Measures

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Primary Outcome Measures :

1. Bone Mineral Density [ Time Frame: 2 years ]
   By Dual-energy x-ray absorptiometry. Results were reported as z-scores as well as as absolute values. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.

Eligibility Criteria

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Ages Eligible for Study:	3 Years to 21 Years (Child, Adult)
Sexes Eligible for Study:	All
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
  • 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

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Sponsors and Collaborators

Indiana University School of Medicine

Investigators

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

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Linda DiMeglio, MD, MD, Indiana University
ClinicalTrials.gov Identifier:	NCT00159419 History of Changes
Other Study ID Numbers:	9902-30
First Posted:	September 12, 2005
Results First Posted:	March 31, 2016
Last Update Posted:	June 14, 2017
Last Verified:	May 2017

Keywords provided by Linda DiMeglio, MD, Indiana University:

Osteogenesis Imperfecta; Fractures; Pediatric; Osteoporosis; Juvenile Pagets

Additional relevant MeSH terms:

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