Long Term Follow-up on Menkes Disease Patients

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Menkes Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hair Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Menkes Kinky Hair Syndrome Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Skin Diseases Skin and Connective Tissue Diseases X-Linked Intellectual Disability

Sex eligibility

All sexes

Study type

Observational

Primary location

Sentynl Therapeutics

Solana Beach, California, 92117, United States

About this study

Primary Objective:

To provide additional clinical and safety data on long term following administration of Copper Histidinate treatment in patients treated with Copper Histidinate under Protocols 09-CH-0059 and 90-CH0149 and untreated patients previously identified and/or enrolled in Protocol 09-CH-0059.
To provide additional Historical Control data on Menkes disease patients.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Long Term Follow-Up

Drug

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.

Other names: CUTX-101

Primary outcomes

Long Term Follow-Up on Survival

Time frame: 12/01/2019 - 12/31/2022

The primary outcome measure will be overall survival.

Sponsors and collaborators

Lead sponsor

Sentynl Therapeutics, Inc.

Industry

Registry information

Official study title

Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients

Important dates

Study start: 2019
Primary completion: 2026
Study completion: 2026
First posted: Apr 8, 2020
Registry last updated: Mar 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Active, Not Recruiting