Copper Histidinate Treatment for Menkes Disease

February 26, 2026 updated by: Sentynl Therapeutics, Inc.
This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • Sacramento, California, United States, 95817
        • University of California Davis Medical Center
      • San Diego, California, United States, 92123
        • Rady Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Connecticut Children's Medical Center
    • Florida
      • Hollywood, Florida, United States, 33021
        • Memorial Healthcare Systems
      • St. Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Lurie Children's Hospital of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Stead Family Children's Hospital
    • Maine
      • Portland, Maine, United States, 04102
        • Maine Medical Center
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Helen DeVos Children's Hospital
    • Montana
      • Helena, Montana, United States, 59601
        • Shodair Children's Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Health Care System
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 6 years (Child)

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sentynl Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

August 27, 2019

First Submitted That Met QC Criteria

August 29, 2019

First Posted (Actual)

August 30, 2019

Study Record Updates

Last Update Posted (Actual)

March 2, 2026

Last Update Submitted That Met QC Criteria

February 26, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CYP-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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