Long Term Follow-up on Menkes Disease Patients | NCT04337684

Studies
Long Term Follow-up on Menkes Disease Patients

NCT04337684

Sponsor

Sentynl Therapeutics, Inc.

Status

Active, not recruiting

Last Update Posted

Mar 2, 2026Actual

Enrollment

50Estimated

Phase

Not provided

Conditions

Menkes Disease

Interventions

Long Term Follow-Up

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2026

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 1, 2019Actual

Primary Completion Date

Feb 26, 2026Actual

Completion Date

Aug 31, 2026Estimated

First Submitted Date

Apr 1, 2020

First Submission Date that Met QC Criteria

Apr 3, 2020

First Posted Date

Apr 8, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 26, 2026

Last Update Posted Date

Mar 2, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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:

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

50Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Historical Control
Treated with Copper Histidinate			Drug: Long Term Follow-Up

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Long Term Follow-Up	Drug	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.

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Any subject previously identified and/or enrolled in either Protocols 09-CH-0059 or 90-CH-0149.
Any new untreated Menkes disease patient born after 1999.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Sentynl Therapeutics		Solana Beach	California	92117	United States

IPD Sharing Statement Module

Plan to Share IPD

Undecided

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D007706	Menkes Kinky Hair Syndrome

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

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Browse Branches

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