Menkes DiseaseNCT04074512
Copper Histidinate Treatment for Menkes Disease
A study testing copper histidinate in people with Menkes Disease, run by Sentynl Therapeutics, Inc. at 20 sites in United States.
Trial summary
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age. Read more about this study
- Who is it for?
- Ages 0 to 6 years
- Conditions the study is for
- Menkes Disease
- What is being tested?
- copper histidinate
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 20 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 6 years
- All sexes
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Emory University
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Duke University Hospital
Durham, North Carolina, 27710
Cook Children's Health Care System
Fort Worth, Texas, 76104
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Shodair Children's Hospital
Helena, Montana, 59601
Memorial Healthcare Systems
Hollywood, Florida, 33021
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 04102
Oregon Health & Science University
Portland, Oregon, 97239
University of California Davis Medical Center
Sacramento, California, 95817
Rady Children's Hospital
San Diego, California, 92123
Seattle Children's Hospital
Seattle, Washington, 98105
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
About this study
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.
- Conditions
- Menkes Disease
- Being tested
- copper histidinate
- Medical subject headings
- Menkes Kinky Hair Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
copper histidinateDrug
Also called CUTX-101
copper histidinate for subcutaneous injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Menkes DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Sentynl Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04074512
- Sponsor's protocol ID
- CYP-001
- Status last verified
- Feb 2026
- First submitted
- 27 Aug 2019
- Last updated
- 2 Mar 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Copper Histidinate Treatment for Menkes Disease
- ClinicalTrials.gov ID
- NCT04074512
- Acronym / sponsor ID
- CYP-001
- Conditions
- Menkes Disease
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Sentynl Therapeutics, Inc.Industry (pharma, biotech, devices)
- First posted
- 30 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.
Study design
Who may qualify
- Ages
- 0 to 6 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| copper histidinateAlso: CUTX-101copper histidinate for subcutaneous injection | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing copper histidinate in people with Menkes Disease, run by Sentynl Therapeutics, Inc. at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2026.