Long Term Follow-up on Menkes Disease Patients

Cyprium Therapeutics

Status: Invitation-only

Conditions: Menkes Disease

Treatments: Drug: Long Term Follow-Up

Study type: Observational

Funder types: Industry

Identifiers

NCT04337684

CYP-002

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

50 participants in 2 patient groups

Historical Control

Treated with Copper Histidinate

Treatment:

Drug: Long Term Follow-Up