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Long Term Follow-up on Menkes Disease Patients
Sponsor: Sentynl Therapeutics, Inc.
Summary
This study will collect long term follow-up data regarding overall survival and neurological parameters from patients previously identified and/or enrolled in Protocols 09-CH-0059 and 90-CH0149 and Historical Control data on Menkes disease patients.
Official title: Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2019-12-01
Completion Date
2026-08-31
Last Updated
2026-03-02
Healthy Volunteers
Not specified
Conditions
Interventions
Long Term Follow-Up
Menkes disease is a form of inherited copper deficiency associated with neurodevelopmental delays and neurological problems. Copper Histidinate is being evaluated for efficacy and safety in patients with Menkes disease. The purpose of this protocol is to collect long term follow-up data. During the study, patients and/or parent/legal guardian will be contacted by the investigator by either telephone or an in-person visit to assess long term follow-up.
Locations (1)
Sentynl Therapeutics
Solana Beach, California, United States