Copper Histidinate Treatment for Menkes Disease

• Conditions: Menkes Disease

• Registration Number: NCT04074512
• Lead Sponsor: Sentynl Therapeutics, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-08-27
• Study First Submitted QC: 2019-08-29
• Study First Posted: 2019-08-30
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-26
• Last Update Posted: 2025-07-01

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified

Trial Locations

Locations (17)

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

University of California Davis Medical Center; 🇺🇸 Sacramento, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, 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

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