Copper Histidinate Treatment for Menkes Disease
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04074512 |
Expanded Access Status :
Available
First Posted : August 30, 2019
Last Update Posted : March 8, 2022
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Condition or disease | Intervention/treatment |
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Menkes Disease | Drug: Copper Histidinate |
Study Type : | Expanded Access |
Expanded Access Type : | Intermediate-size Population |
Official Title: | Copper Histidinate Treatment for Menkes Disease |

- Drug: Copper Histidinate
Restoring Copper Histidinate levels normally found in the blood to assist in the maintenance of copper homeostasis.Other Name: CUTX-101

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Ages Eligible for Study: | 0 Years to 6 Years (Child) |
Sexes Eligible for Study: | All |
Inclusion Criteria:
- The subject must be a newly diagnosed Menkes disease patient in the United States.
- 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.
- 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).
- For newly diagnosed Menkes disease patients, whose molecular ATP7A gene mutation confirmation is pending these patients should have serum copper levels < 75 mcg/dL.
- Ability to adhere to the prescribed subcutaneous Copper Histidinate injection regimen.
- Willingness to comply with all study visits and procedures.
Exclusion Criteria:
- Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease (e.g., serum creatinine >1.0 mg/dL).
- History of bleeding diatheses.
- 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.
- Participation in any other investigational trial in which receipt of investigational drug or device occurred within 30 days prior to screening for this study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04074512
Contact: Cyprium Study Team | (212) 574-2851 | studyinfo@cypriumtx.com |
Responsible Party: | Cyprium Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04074512 |
Other Study ID Numbers: |
CYP-001 |
First Posted: | August 30, 2019 Key Record Dates |
Last Update Posted: | March 8, 2022 |
Last Verified: | March 2022 |
Menkes Kinky Hair Syndrome Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Mental Retardation, X-Linked Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Genetic Diseases, X-Linked |
Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Hair Diseases Skin Diseases Metabolic Diseases Copper Trace Elements Micronutrients Physiological Effects of Drugs |