Copper Histidinate Treatment for Menkes Disease

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Conditions studied: Menkes Disease

In brief

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

Key facts

Study ID

NCT04074512

Run by

Sentynl Therapeutics, Inc.

Last updated by the study team

2026-03-02

Who can join

Age: any, up to 6. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of California Davis Medical Center — Sacramento, California, United States
• Rady Children's Hospital — San Diego, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• Memorial Healthcare Systems — Hollywood, Florida, United States
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
• Emory University — Atlanta, Georgia, United States
• Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States
• Maine Medical Center — Portland, Maine, United States
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
• Shodair Children's Hospital — Helena, Montana, United States
• Duke University Hospital — Durham, North Carolina, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Cook Children's Health Care System — Fort Worth, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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