Trial Outcomes & Findings for Bisphosphonate Therapy for Osteogenesis Imperfecta (NCT NCT00159419)

NCT ID: NCT00159419

Last Updated: 2017-06-14

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 18 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2017-06-14

Participant Flow

Recruited from clinics

Participant milestones

Measure	Pamidronate Treatment	Alendronate Treatment
Overall Study STARTED	9	9
Overall Study COMPLETED	9	9
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Bisphosphonate Therapy for Osteogenesis Imperfecta

Baseline characteristics by cohort

Measure	Pamidronate Treatment n=9 Participants	Alendronate Treatment n=9 Participants	Total n=18 Participants Total of all reporting groups
Age, Categorical <=18 years	9 Participants n=5 Participants	9 Participants n=7 Participants	18 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	8.4 years n=5 Participants	9.0 years n=7 Participants	8.7 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants	9 participants n=7 Participants	18 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Pamidronate Treatment n=9 Participants	Alendronate n=9 Participants
Bone Mineral Density	-1.3 z-score Standard Deviation 2.0	-1.1 z-score Standard Deviation 1.2

Adverse Events

Pamidronate Treatment

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Alendronate Treatment

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Pamidronate Treatment n=9 participants at risk Acute phase reaction with infusion	Alendronate Treatment n=9 participants at risk
General disorders Acute Phase Reaction	77.8% 7/9 • Number of events 8	0.00% 0/9

Additional Information

Dr. Linda DiMeglio

Indiana University School of Medicine

Phone: 317-944-3889

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place