Long Term Follow-up on Menkes Disease Patients

• Conditions: Menkes Disease
• Interventions: Drug: Long Term Follow-Up

• Registration Number: NCT04337684
• Lead Sponsor: Cyprium Therapeutics, Inc.

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.

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.

Study Timeline

• Study Start: 2019-12-01
• Study First Submitted: 2020-04-01
• Study First Submitted QC: 2020-04-03
• Study First Posted: 2020-04-08
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-26
• Last Update Posted: 2023-09-28
• Primary Completion: 2023-12-31
• Study Completion: 2023-12-31

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 50

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Treated with Copper Histidinate	Long Term Follow-Up	-

Primary Outcome Measures

Name	Time	Method
Long Term Follow-Up on Survival	12/01/2019 - 12/31/2022	The primary outcome measure will be overall survival.

Trial Locations

Locations (1)

Cyprium Study Team; 🇺🇸 New York, New York, United States