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Copper Histidine Therapy for Menkes Diseases

This study has been completed.
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) )
ClinicalTrials.gov Identifier:
NCT00001262
First received: November 3, 1999
Last updated: September 29, 2015
Last verified: September 2015
Results First Received: August 5, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Kinky Hair Syndrome
Intervention: Drug: Copper Histidine

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Early Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Late Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Mild Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms

Participant Flow:   Overall Study
    Early     Late     Mild  
STARTED     35     22     3  
COMPLETED     25     8     3  
NOT COMPLETED     10     14     0  
Death                 10                 11                 0  
Withdrawal by Subject                 0                 3                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Early Classic Menkes disease: Copper histidine treatment beginning within 1 month of age
Late Classic Menkes disease: Copper histidine treatment beginning after 1 month of age and after onset of symptoms
Mild Milder variants of Menkes disease: Copper histidine treatment beginning late (L) and after onset of (milder) symptoms
Total Total of all reporting groups

Baseline Measures
    Early     Late     Mild     Total  
Number of Participants  
[units: participants]
  35     22     3     60  
Age  
[units: Participants]
       
<=18 years     35     22     3     60  
Between 18 and 65 years     0     0     0     0  
>=65 years     0     0     0     0  
Gender  
[units: Participants]
       
Female     1     0     0     1  
Male     34     22     3     59  
Ethnicity (NIH/OMB)  
[units: Participants]
       
Hispanic or Latino     3     0     0     3  
Not Hispanic or Latino     30     21     3     54  
Unknown or Not Reported     2     1     0     3  
Race (NIH/OMB)  
[units: Participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     1     0     1  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     9     1     0     10  
White     22     19     3     44  
More than one race     1     0     0     1  
Unknown or Not Reported     3     1     0     4  



  Outcome Measures
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1.  Primary:   Gross Motor Development at 36 Mos of Age or at Death (Mos)   [ Time Frame: 36 months or death ]

2.  Primary:   Fine Motor Adaptive Development at 36 Mos of Age or at Death (Mos)   [ Time Frame: 36 months or death ]

3.  Primary:   Personal-Social Development at 36 Mos of Age or at Death (Mos)   [ Time Frame: 36 months or death ]

4.  Primary:   Language Development at 36 Mos of Age or at Death (Mos)   [ Time Frame: 36 months or death ]

5.  Secondary:   Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Weight Percentile   [ Time Frame: 36 months or death ]

6.  Secondary:   Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Length Percentile   [ Time Frame: 36 months or death ]

7.  Secondary:   Somatic Growth Percentiles at 3 Years of Age (or at Age of Death) - Head Circumference Percentile   [ Time Frame: 36 months or death ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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
e-mail: kalers@mail.nih.gov


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
Study First Received: November 3, 1999
Results First Received: August 5, 2014
Last Updated: September 29, 2015
Health Authority: United States: Federal Government
United States: Food and Drug Administration