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Trial record 2 of 4 for:    Menkes Disease

Copper Histidine Therapy for Menkes Diseases

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ClinicalTrials.gov Identifier: NCT00001262
Recruitment Status : Completed
First Posted : November 4, 1999
Results First Posted : October 30, 2015
Last Update Posted : October 30, 2015
Sponsor:
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 Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Kinky Hair Syndrome
Intervention Drug: Copper Histidine
Enrollment 60
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Early Late Mild
Hide Arm/Group Description Classic Menkes disease: Copper histidine treatment beginning within 1 month of age Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Period Title: Overall Study
Started 35 22 3
Completed 25 8 3
Not Completed 10 14 0
Reason Not Completed
Death             10             11             0
Withdrawal by Subject             0             3             0
Arm/Group Title Early Late Mild Total
Hide Arm/Group Description Classic Menkes disease: Copper histidine treatment beginning within 1 month of age Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms Total of all reporting groups
Overall Number of Baseline Participants 35 22 3 60
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 22 participants 3 participants 60 participants
<=18 years
35
 100.0%
22
 100.0%
3
 100.0%
60
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 22 participants 3 participants 60 participants
Female
1
   2.9%
0
   0.0%
0
   0.0%
1
   1.7%
Male
34
  97.1%
22
 100.0%
3
 100.0%
59
  98.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 22 participants 3 participants 60 participants
Hispanic or Latino
3
   8.6%
0
   0.0%
0
   0.0%
3
   5.0%
Not Hispanic or Latino
30
  85.7%
21
  95.5%
3
 100.0%
54
  90.0%
Unknown or Not Reported
2
   5.7%
1
   4.5%
0
   0.0%
3
   5.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 22 participants 3 participants 60 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
   4.5%
0
   0.0%
1
   1.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
9
  25.7%
1
   4.5%
0
   0.0%
10
  16.7%
White
22
  62.9%
19
  86.4%
3
 100.0%
44
  73.3%
More than one race
1
   2.9%
0
   0.0%
0
   0.0%
1
   1.7%
Unknown or Not Reported
3
   8.6%
1
   4.5%
0
   0.0%
4
   6.7%
1.Primary Outcome
Title Gross Motor Development at 36 Mos of Age or at Death (Mos)
Hide Description This was measured based on the Denver Developmental Screening Test (DDST) I or II for age-appropriate gross motor development in apparently normal healthy subjects at specific ages (in months). The DDST employs a grid to assess expected developmental milestones in relation to chronologic age.
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - months
13.743  (12.200) 2.455  (2.154) 15.667  (9.815)
2.Primary Outcome
Title Fine Motor Adaptive Development at 36 Mos of Age or at Death (Mos)
Hide Description This was measured based on the Denver Developmental Screening Test (DDST) I or II for age-appropriate fine motor development in apparently normal healthy subjects at specific ages (in months). The DDST employs a grid to assess expected developmental milestones in relation to chronologic age.
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Months
16.200  (12.762) 2.409  (1.652) 17.667  (13.204)
3.Primary Outcome
Title Personal-Social Development at 36 Mos of Age or at Death (Mos)
Hide Description This was measured based on the Denver Developmental Screening Test (DDST) I or II for age-appropriate personal-social development in apparently normal healthy subjects at specific ages (in months). The DDST employs a grid to assess expected developmental milestones in relation to chronologic age.
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Months
17.657  (13.482) 3.364  (3.499) 17.667  (15.308)
4.Primary Outcome
Title Language Development at 36 Mos of Age or at Death (Mos)
Hide Description This was measured based on the Denver Developmental Screening Test (DDST) I or II for age-appropriate language development in apparently normal healthy subjects at specific ages (in months). The DDST employs a grid to assess expected developmental milestones in relation to chronologic age.
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Months
15.800  (12.034) 3.227  (2.943) 21.000  (9.539)
5.Secondary Outcome
Title Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Weight Percentile
Hide Description [Not Specified]
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Percentile
12.086  (19.589) 11.273  (17.097) 5.000  (8.660)
6.Secondary Outcome
Title Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Length Percentile
Hide Description [Not Specified]
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Percentile
8.286  (13.501) 15.455  (23.192) 28.333  (40.723)
7.Secondary Outcome
Title Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Head Circumference Percentile
Hide Description [Not Specified]
Time Frame 36 months or death
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Early Late Mild
Hide Arm/Group Description:
Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Overall Number of Participants Analyzed 35 22 3
Mean (Standard Deviation)
Unit of Measure: Other - Percentile
33.286  (27.060) 11.136  (14.551) 18.333  (27.538)
Time Frame 36 months or death
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Early Late Mild
Hide Arm/Group Description Classic Menkes disease: Copper histidine treatment beginning within 1 month of age Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
All-Cause Mortality
Early Late Mild
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Early Late Mild
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   10/35 (28.57%)      11/22 (50.00%)      0/3 (0.00%)    
Nervous system disorders       
Death [1]  10/35 (28.57%)  10 11/22 (50.00%)  11 0/3 (0.00%)  0
Dehydration  1/35 (2.86%)  1 0/22 (0.00%)  0 0/3 (0.00%)  0
[1]
Menkes disease
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Early Late Mild
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/35 (0.00%)      0/22 (0.00%)      0/3 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Kaler, Stephen G
Organization: National Institute of Child Health and Human Development
Phone: +1 301 451 6034
Publications of Results:
Responsible Party: National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )
ClinicalTrials.gov Identifier: NCT00001262     History of Changes
Other Study ID Numbers: 900149
90-CH-0149
First Submitted: November 3, 1999
First Posted: November 4, 1999
Results First Submitted: August 5, 2014
Results First Posted: October 30, 2015
Last Update Posted: October 30, 2015