Copper Histidinate Treatment for Menkes Disease
1 other identifier
expanded_access
N/A
1 country
20
Brief Summary
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 27, 2019
CompletedFirst Posted
Study publicly available on registry
August 30, 2019
CompletedMarch 2, 2026
February 1, 2026
August 27, 2019
February 26, 2026
Conditions
Interventions
Eligibility Criteria
You may qualify if:
- The subject must have been treated in protocol 09-CH-0059 or must be a newly diagnosed Menkes disease patient in the US.
- Parent or legal guardian is willing and able to sign and date an informed consent form.
- Male or female, aged 0 to \<6 years of age.
- Diagnosis of Menkes disease: A confirmed gene mutation in ATP7A is not required; however, the test must be at least pending prior to initiating therapy. For those patients whose molecular ATP7A gene mutation confirmation is pending, they should have serum copper \<75 mcg/dL. If the mutation is subsequently not confirmed, the patient should discontinue treatment.
- Ability to adhere to the prescribed subcutaneous copper histidinate injection regimen.
- Willingness to adhere to all recommended visits and procedures.
- Resident of the US.
You may not qualify if:
- Diagnosis of Wilson disease.
- Any disease or condition that, in the opinion of the Investigator, has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements.
- Receipt of concomitant medication or device not approved for any use (i.e. experimental) by FDA within 30 days prior to screening for this study, except for participation in protocol 09-CH-0059.
- Patient/caregiver cannot or will not appropriately comply with protocol recommendations (e.g., site visits, renal monitoring).
- Currently receiving any concomitant copper-containing medications.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Memorial Healthcare Systems
Hollywood, Florida, 33021, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
Emory University
Atlanta, Georgia, 30322, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
Maine Medical Center
Portland, Maine, 04102, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Shodair Children's Hospital
Helena, Montana, 59601, United States
Duke University Hospital
Durham, North Carolina, 27710, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Cook Children's Health Care System
Fort Worth, Texas, 76104, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 27, 2019
First Posted
August 30, 2019
Last Updated
March 2, 2026
Record last verified: 2026-02