Long Term Follow-up on Menkes Disease Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 9 October 2023
• Main ID: NCT04337684
• Date of registration: 01/04/2020
• Prospective Registration: No
• Primary sponsor: Cyprium Therapeutics, Inc.
• Public title: Long Term Follow-up on Menkes Disease Patients
• Scientific title: Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
• Date of first enrolment: December 1, 2019
• Target sample size: 50
• Recruitment status: Enrolling by invitation
• URL: https://clinicaltrials.gov/ct2/show/NCT04337684
• Study type: Observational

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- The subject must have been previously identified and/or enrolled under Protocols
09-CH-0059 or 90-CH-0149; or An untreated Menkes disease patients for whom data
collection is incomplete under Protocols 09-CH-0059 (Amendment M); or Other untreated
Menkes disease patients.

- 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. If the patient is > 18 years of age,
the patient must sign the informed consent.

- Male or female, aged 0 to < 65 years of age.

Exclusion Criteria:

- Unwillingness/unable to participate in the study.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Menkes Disease

Intervention(s)

Drug: Long Term Follow-Up

Primary Outcome(s)

Long Term Follow-Up on Survival [Time Frame: 12/01/2019 - 12/31/2022]

Secondary ID(s)

CYP-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors