Long Term Follow-up on Menkes Disease Patients

Menkes Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04337684
• Other study ID #: CYP-002
• Status: Enrolling by invitation
• Start date: December 1, 2019
• Est. completion date: December 31, 2023

Study information

• Verified date: September 2023
• Source: Cyprium Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 50
• Est. completion date: December 31, 2023
• Est. primary completion date: December 31, 2023
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Menkes Disease

Intervention

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.

Locations

Country	Name	City	State
United States	Cyprium Study Team	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Cyprium Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long Term Follow-Up on Survival	The primary outcome measure will be overall survival.	12/01/2019 - 12/31/2022