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Copper Histidinate Treatment for Menkes Disease

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 : August 30, 2019
Sponsor:
Information provided by (Responsible Party):
Cyprium Therapeutics, Inc.

Study Description
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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
Menkes Disease Drug: Copper Histidinate

Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: Copper Histidinate Treatment for Menkes Disease

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: Copper Histidinate
    Restoring Copper Histidinate levels normally found in the blood to assist in the maintenance of copper homeostasis.
    Other Name: CUTX-101
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   up to 6 Years   (Child)
Sexes Eligible for Study:   All
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Cyprium Study Team (212) 574-2851 studyinfo@cypriumtx.com

Sponsors and Collaborators
Cyprium Therapeutics, Inc.
More Information
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Additional Information:
Related Info  This link exits the ClinicalTrials.gov site

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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: August 30, 2019
Last Verified: August 2019
Additional relevant MeSH terms:
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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
Nutrients
Growth Substances
Physiological Effects of Drugs