Long Term Follow-up on Menkes Disease Patients

NCT04337684 | Active Not Recruiting FDA Drug

• 1 other identifier: CYP-002
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Menkes Disease

Outcome Measures

Primary Outcomes (1)

• Long Term Follow-Up on Survival

  The primary outcome measure will be overall survival.

  12/01/2019 - 12/31/2022

Study Arms (2)

Historical Control

Treated with Copper Histidinate

Drug: Long Term Follow-Up

Interventions

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.

Also known as: CUTX-101

Treated with Copper Histidinate

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

1. Any subject previously identified and/or enrolled in either Protocols 09-CH-0059 or 90-CH-0149. 2. Any new untreated Menkes disease patient born after 1999.

You may qualify if:

• 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.

You may not qualify if:

• \- Unwillingness/unable to participate in the study.

Sponsors & Collaborators

• Sentynl Therapeutics, Inc.: lead

Study Sites (1)

Sentynl Therapeutics

Solana Beach, California, 92117, United States

Location

MeSH Terms

Conditions

Menkes Kinky Hair Syndrome

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 1, 2020
• First Posted: April 8, 2020
• Study Start: December 1, 2019
• Primary Completion: February 26, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: March 2, 2026

Record last verified: 2026-02

Locations

US (1)