Copper Histidinate Treatment for Menkes Disease

Sentynl Therapeutics

Status

Conditions: Menkes Disease

Treatments: Drug: copper histidinate

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT04074512

CYP-001

Details and patient eligibility

About

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

Sex: All

Ages: Under 6 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.