Researchers track menkes disease over time to unlock key insights
NCT ID NCT04337684
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study gathers long-term health information from up to 50 people with Menkes disease, a rare genetic disorder. The goal is to track survival and nervous system changes over time. No new treatments are being tested; instead, researchers are collecting data to better understand the disease's natural course.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 50 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2019
- Expected to finish
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Aug 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
1. Any subject previously identified and/or enrolled in either Protocols 09-CH-0059 or 90-CH-0149. 2. Any new untreated Menkes disease patient born after 1999.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Sentynl Therapeutics
Solana Beach, California, 92117, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.