NCT04337684

Long Term Follow-up on Menkes Disease Patients

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Sentynl Therapeutics, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: —

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.

Detailed description

Primary Objective: 1. 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. 2. To provide additional Historical Control data on Menkes disease patients.

Conditions

• Menkes Disease

Interventions

Type	Name	Description
DRUG	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.

Timeline

• Start date: 2019-12-01
• Primary completion: 2026-02-26
• Completion: 2026-08-31
• First posted: 2020-04-08
• Last updated: 2026-03-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study