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.
COMPLETED
PHASE3
34 participants
Baseline vs. 12 months after first dose
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=99 Participants
|
15 Participants
n=107 Participants
|
34 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
30 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
27 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Bone Mineral Density in Response to Pamidronate
|
-5.01 Z-score
STANDARD_DEVIATION 1.79 • n=99 Participants
|
-5.68 Z-score
STANDARD_DEVIATION 0.96 • n=107 Participants
|
-5.32 Z-score
STANDARD_DEVIATION 1.47 • n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline vs. 12 months after first dosePopulation: 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 dosePopulation: 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 dosePopulation: 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 dosePopulation: 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 dosePopulation: 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 dosePopulation: 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
Active Comparator: 2
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
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place