Menkes Disease
Conditions
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.
Interventions
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.
Sponsors
Sentynl Therapeutics, Inc.
Study design
Observational model
CASE_CONTROL
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Long Term Follow-Up on Survival | 12/01/2019 - 12/31/2022 | The primary outcome measure will be overall survival. |
Countries
United States
Outcome results
None listed