Long Term Follow-up on Menkes Disease Patients

September 26, 2023 updated by: Cyprium Therapeutics, Inc.

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10014
        • Cyprium Study Team

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Control
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Historical Control
Treated with Copper Histidinate
Drug: Long Term Follow-Up

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.

Other Names:
  • CUTX-101

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cyprium Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2019

Primary Completion (Estimated)

December 31, 2023

Study Completion (Estimated)

December 31, 2023

Study Registration Dates

First Submitted

April 1, 2020

First Submitted That Met QC Criteria

April 3, 2020

First Posted (Actual)

April 8, 2020

Study Record Updates

Last Update Posted (Actual)

September 28, 2023

Last Update Submitted That Met QC Criteria

September 26, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CYP-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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