Copper Histidinate Treatment for Menkes Disease
Study Details
Study Description
Brief Summary
This study investigates the clinical effects and safety of subcutaneous Copper Histidinate treatment for newly diagnosed Menkes disease patients under 6 years of age.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Inclusion Criteria:
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The subject must be a newly diagnosed Menkes disease patient in the United States.
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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.
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Male or female, aged 0 to <6 years of age.
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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).
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For newly diagnosed Menkes disease patients, whose molecular ATP7A gene mutation confirmation is pending these patients should have serum copper levels < 75 mcg/dL.
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Ability to adhere to the prescribed subcutaneous Copper Histidinate injection regimen.
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Willingness to comply with all study visits and procedures.
Exclusion Criteria:
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Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease (e.g., serum creatinine >1.0 mg/dL).
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History of bleeding diatheses.
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Diagnosis of Wilson disease.
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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.
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Participation in any other investigational trial in which receipt of investigational drug or device occurred within 30 days prior to screening for this study.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Cyprium Therapeutics, Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- CYP-001