APPROVED_FOR_MARKETINGNot Applicable

Copper Histidinate Treatment for Menkes Disease

United States

Plain-language summary

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

Age range0 Years – 6 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.

NCT IDNCT04074512

SponsorSentynl Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

Who can participate

Age range0 Years – 6 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.