Menkes DiseaseNCT04337684
Long Term Follow-up on Menkes Disease Patients
An observational study of Menkes Disease, run by Sentynl Therapeutics, Inc. at 1 site in United States.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Menkes Disease
- What is being tested?
- Long Term Follow-Up
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2019to 31 Aug 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- Unwillingness/unable to participate in the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Sentynl Therapeutics
Solana Beach, California, 92117
About this study
Official title: Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
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.
Read the detailed description
Primary Objective:
- 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.
- To provide additional Historical Control data on Menkes disease patients.
- Conditions
- Menkes Disease
- Being tested
- Long Term Follow-Up
- Medical subject headings
- Menkes Kinky Hair Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Long Term Follow-UpDrug
Also called CUTX-101
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.
| Study group | Role | What they receive |
|---|---|---|
| Historical Control | Not described | |
| Treated with Copper Histidinate | Not described Receives: Drug: Long Term Follow-Up |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long Term Follow-Up on Survival
The primary outcome measure will be overall survival.
Measured: 12/01/2019 - 12/31/2022
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2020
- Study start1 Dec 2019
- Primary completion26 Feb 2026
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Menkes DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sentynl Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04337684
- Sponsor's protocol ID
- CYP-002
- Status last verified
- Feb 2026
- First submitted
- 1 Apr 2020
- Last updated
- 2 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
- ClinicalTrials.gov ID
- NCT04337684
- Acronym / sponsor ID
- CYP-002
- Conditions
- Menkes Disease
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Sentynl Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Dec 2019 (actual)
- Primary completion
- 26 Feb 2026 (actual)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 8 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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.
- To provide additional Historical Control data on Menkes disease patients.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Historical Control | – | – |
| Treated with Copper Histidinate | – | Drug: Long Term Follow-Up |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- Unwillingness/unable to participate in the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Long Term Follow-UpAlso: CUTX-101Menkes 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. | Drug | Treated with Copper Histidinate |
Primary outcome
Long Term Follow-Up on Survival
Time frame: 12/01/2019 - 12/31/2022
The primary outcome measure will be overall survival.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Menkes Disease, run by Sentynl Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long Term Follow-Up on Survival (time frame: 12/01/2019 - 12/31/2022). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2026.