Copper Histidinate Treatment for Menkes Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 22 April 2024
• Main ID: NCT04074512
• Date of registration: 27/08/2019
• Prospective Registration: Yes
• Primary sponsor: Sentynl Therapeutics, Inc.
• Public title: Copper Histidinate Treatment for Menkes Disease
• Scientific title: Copper Histidinate Treatment for Menkes Disease
• Date of first enrolment: December 2, 2019
• Recruitment status: Available
• URL: https://clinicaltrials.gov/ct2/show/NCT04074512
• Study type: Expanded Access

Contacts

• Name: Sentynl Therapeutics Study Team

Key inclusion & exclusion criteria

Inclusion Criteria:

1. The subject must be a newly diagnosed Menkes disease patient in the United States.

2. Must sign and date an informed consent form by parent or legal guardian for this study
prior to any assessment being done in this study.

3. Male or female, aged 0 to <6 years of age.

4. Confirmed diagnosis of Menkes disease based on the following clinical and/or
biochemical and/or molecular characteristics:

Clinical: Abnormal hair color and/or texture, and/or seizures, and/or hypotonia,
and/or developmental delay; or Biochemical: Low serum copper levels (< 75 mcg/dL)
and/or ceruloplasmin and/or abnormal plasma catecholamine levels; or Molecular:
Mutation(s) in the ATP7A gene (deletion/duplication, nonsense, missense, or canonical
or non-canonical splice junction mutations).

5. For newly diagnosed Menkes disease patients, whose molecular ATP7A gene mutation
confirmation is pending these patients should have serum copper levels < 75 mcg/dL.

6. Ability to adhere to the prescribed subcutaneous Copper Histidinate injection regimen.

7. Willingness to comply with all study visits and procedures.

Exclusion Criteria:

1. Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease
(e.g., serum creatinine >1.0 mg/dL).

2. History of bleeding diatheses.

3. Diagnosis of Wilson disease.

4. 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.

5. Participation in any other investigational trial in which receipt of investigational
drug or device occurred within 30 days prior to screening for this study.



Age minimum: 0 Years
Age maximum: 6 Years
Gender: All

Health Condition(s) or Problem(s) studied

Menkes Disease

Intervention(s)

Drug: Copper Histidinate

Secondary ID(s)

CYP-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors