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Pamidronate to Treat Osteogenesis Imperfecta in Children

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ClinicalTrials.gov Identifier: NCT00005901
Recruitment Status : Completed
First Posted : June 7, 2000
Results First Posted : March 1, 2016
Last Update Posted : March 1, 2016
Sponsor:
Collaborator:
National Institutes of Health Clinical Center (CC)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Osteogenesis Imperfecta
Intervention Drug: Pamidronate (Aredia)
Enrollment 34
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Active Comparator: 1 Active Comparator: 2
Hide Arm/Group Description Receives treatment every 3 months Receives treatment every 6 months.
Period Title: Overall Study
Started 19 15
Completed 19 15
Not Completed 0 0
Arm/Group Title Active Comparator: 1 Active Comparator: 2 Total
Hide Arm/Group Description Subjects who received Pamidronate every 3 months for 3 years. Subjects who received Pamidronate every 6 months for 3 years. Total of all reporting groups
Overall Number of Baseline Participants 19 15 34
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 15 participants 34 participants
<=18 years
19
 100.0%
15
 100.0%
34
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 15 participants 34 participants
Female
8
  42.1%
9
  60.0%
17
  50.0%
Male
11
  57.9%
6
  40.0%
17
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 15 participants 34 participants
Hispanic or Latino
3
  15.8%
1
   6.7%
4
  11.8%
Not Hispanic or Latino
16
  84.2%
14
  93.3%
30
  88.2%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 15 participants 34 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   5.3%
1
   6.7%
2
   5.9%
White
14
  73.7%
13
  86.7%
27
  79.4%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
4
  21.1%
1
   6.7%
5
  14.7%
Bone Mineral Density in Response to Pamidronate   [1] 
Mean (Standard Deviation)
Unit of measure:  Z-score
Number Analyzed 19 participants 15 participants 34 participants
-5.01  (1.79) -5.68  (0.96) -5.32  (1.47)
[1]
Measure Description: 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.
1.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 12 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-3.54  (1.51) -4.98  (1.16)
2.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 18 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-3.71  (1.4) -4.65  (1.06)
3.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 24 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-3.48  (1.31) -4.7  (1.37)
4.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 30 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-3.48  (1.09) -4.05  (1.28)
5.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 36 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-3.31  (1.22) -4.18  (1.67)
6.Primary Outcome
Title Change in Bone Mineral Density in Response to Pamidronate
Hide Description 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.
Time Frame Baseline vs. 6 months after first dose
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects received Pamidronate for 3 years.
Arm/Group Title Pamidronate Every 3 Months for 3 Years Pamidronate Every 6 Months for 3 Years
Hide Arm/Group Description:
Subjects who received Pamidronate every 3 months for 3 years.
Subjects who received Pamidronate every 6 months for 3 years.
Overall Number of Participants Analyzed 19 15
Mean (Standard Deviation)
Unit of Measure: Z-score
-4.01  (1.79) -4.98  (1.15)
Time Frame 3 years.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Active Comparator: 1 Active Comparator: 2
Hide Arm/Group Description Subjects who received Pamidronate every 3 months for 3 years. Subjects who received Pamidronate every 6 months for 3 years.
All-Cause Mortality
Active Comparator: 1 Active Comparator: 2
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Active Comparator: 1 Active Comparator: 2
Affected / at Risk (%) Affected / at Risk (%)
Total   0/19 (0.00%)   0/15 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Active Comparator: 1 Active Comparator: 2
Affected / at Risk (%) Affected / at Risk (%)
Total   0/19 (0.00%)   1/15 (6.67%) 
Vascular disorders     
Hypertension   0/19 (0.00%)  1/15 (6.67%) 
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Joan Marini
Organization: National Institute of Child Health and Human Development
Phone: +1 301 594 3418
Responsible Party: National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )
ClinicalTrials.gov Identifier: NCT00005901     History of Changes
Other Study ID Numbers: 000136
00-CH-0136 ( Other Identifier: NIH NICHD IRB )
First Submitted: June 6, 2000
First Posted: June 7, 2000
Results First Submitted: November 24, 2015
Results First Posted: March 1, 2016
Last Update Posted: March 1, 2016