Copper Histidinate Treatment for Menkes Disease

NCT ID: NCT04074512

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.

Detailed Description

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Conditions

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Menkes Disease

Interventions

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copper histidinate

copper histidinate for subcutaneous injection

Intervention Type DRUG

Other Intervention Names

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CUTX-101

Eligibility Criteria

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Inclusion Criteria

1. The subject must have been treated in protocol 09-CH-0059 or must be a newly diagnosed Menkes disease patient in the US.
2. Parent or legal guardian is willing and able to sign and date an informed consent form.
3. Male or female, aged 0 to \<6 years of age.
4. 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.
5. Ability to adhere to the prescribed subcutaneous copper histidinate injection regimen.
6. Willingness to adhere to all recommended visits and procedures.
7. Resident of the US.

Exclusion Criteria

1. Diagnosis of Wilson disease.
2. 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.
3. 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.
4. Patient/caregiver cannot or will not appropriately comply with protocol recommendations (e.g., site visits, renal monitoring).
5. Currently receiving any concomitant copper-containing medications.
Minimum Eligible Age

0 Years

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sentynl Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status AVAILABLE

University of California Davis Medical Center

Sacramento, California, United States

Site Status AVAILABLE

Rady Children's Hospital

San Diego, California, United States

Site Status AVAILABLE

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status AVAILABLE

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Site Status AVAILABLE

Memorial Healthcare Systems

Hollywood, Florida, United States

Site Status AVAILABLE

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status AVAILABLE

Emory University

Atlanta, Georgia, United States

Site Status AVAILABLE

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status AVAILABLE

University of Iowa Stead Family Children's Hospital

Iowa City, Iowa, United States

Site Status AVAILABLE

Maine Medical Center

Portland, Maine, United States

Site Status AVAILABLE

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status AVAILABLE

Shodair Children's Hospital

Helena, Montana, United States

Site Status AVAILABLE

Duke University Hospital

Durham, North Carolina, United States

Site Status AVAILABLE

Oregon Health & Science University

Portland, Oregon, United States

Site Status AVAILABLE

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status AVAILABLE

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status AVAILABLE

Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status AVAILABLE

Cook Children's Health Care System

Fort Worth, Texas, United States

Site Status AVAILABLE

Seattle Children's Hospital

Seattle, Washington, United States

Site Status AVAILABLE

Countries

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United States

Central Contacts

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Sentynl Therapeutics Study Team

Role: CONTACT

Phone: 888-507-5206

Email: [email protected]

Related Links

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Other Identifiers

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CYP-001

Identifier Type: -

Identifier Source: org_study_id