Trial Outcomes & Findings for Pamidronate to Treat Osteogenesis Imperfecta in Children (NCT NCT00005901)

NCT ID: NCT00005901

Last Updated: 2016-03-01

Results Overview

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 34 participants
• Primary outcome timeframe: Baseline vs. 12 months after first dose
• Results posted on: 2016-03-01

Participant Flow

Participant milestones

Measure	Active Comparator: 1 Receives treatment every 3 months	Active Comparator: 2 Receives treatment every 6 months.
Overall Study STARTED	19	15
Overall Study COMPLETED	19	15
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pamidronate to Treat Osteogenesis Imperfecta in Children

Baseline characteristics by cohort

Measure	Active Comparator: 1 n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Active Comparator: 2 n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.	Total n=34 Participants Total of all reporting groups
Age, Categorical <=18 years	19 Participants n=5 Participants	15 Participants n=7 Participants	34 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
Sex: Female, Male Female	8 Participants n=5 Participants	9 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	16 Participants n=5 Participants	14 Participants n=7 Participants	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants	13 Participants n=7 Participants	27 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Bone Mineral Density in Response to Pamidronate	-5.01 Z-score STANDARD_DEVIATION 1.79 • n=5 Participants	-5.68 Z-score STANDARD_DEVIATION 0.96 • n=7 Participants	-5.32 Z-score STANDARD_DEVIATION 1.47 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline vs. 12 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-3.54 Z-score Standard Deviation 1.51	-4.98 Z-score Standard Deviation 1.16

PRIMARY outcome

Timeframe: Baseline vs. 18 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-3.71 Z-score Standard Deviation 1.4	-4.65 Z-score Standard Deviation 1.06

PRIMARY outcome

Timeframe: Baseline vs. 24 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-3.48 Z-score Standard Deviation 1.31	-4.7 Z-score Standard Deviation 1.37

PRIMARY outcome

Timeframe: Baseline vs. 30 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-3.48 Z-score Standard Deviation 1.09	-4.05 Z-score Standard Deviation 1.28

PRIMARY outcome

Timeframe: Baseline vs. 36 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-3.31 Z-score Standard Deviation 1.22	-4.18 Z-score Standard Deviation 1.67

PRIMARY outcome

Timeframe: Baseline vs. 6 months after first dose

Population: All subjects received Pamidronate for 3 years.

Dual-energy X-ray Absorptiometry (DXA) measurements were obtained using a Hologic QDR 4500 densitometer and low density software package. Measurements have a precision of 0.011 SD. Raw measurements were converted to Z-scores for analysis using the manufacturer's reference standards for age and pubertal status.

Outcome measures

Measure	Pamidronate Every 3 Months for 3 Years n=19 Participants Subjects who received Pamidronate every 3 months for 3 years.	Pamidronate Every 6 Months for 3 Years n=15 Participants Subjects who received Pamidronate every 6 months for 3 years.
Change in Bone Mineral Density in Response to Pamidronate	-4.01 Z-score Standard Deviation 1.79	-4.98 Z-score Standard Deviation 1.15

Adverse Events

Active Comparator: 1

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Active Comparator: 2

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Active Comparator: 1 n=19 participants at risk Subjects who received Pamidronate every 3 months for 3 years.	Active Comparator: 2 n=15 participants at risk Subjects who received Pamidronate every 6 months for 3 years.
Vascular disorders Hypertension	0.00% 0/19 • 3 years.	6.7% 1/15 • 3 years.

Additional Information

Joan Marini

National Institute of Child Health and Human Development

Phone: +1 301 594 3418

Email: marinij@cc1.nichd.nih.gov

Results disclosure agreements

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