Clincosm LogoSign in Get a demo

Long Term Follow-up on Menkes Disease Patients

Sponsor
Cyprium Therapeutics, Inc. (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04337684
Collaborator
(none)
50
Enrollment
1
Location
37
Anticipated Duration (Months)
1.4
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Long Term Follow-Up

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 Design

Study Type:
Observational
Anticipated Enrollment :
50 participants
Observational Model:
Case-Control
Time Perspective:
Prospective
Official Title:
Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
Actual Study Start Date :
Dec 1, 2019
Anticipated Primary Completion Date :
Dec 31, 2022
Anticipated Study Completion Date :
Dec 31, 2022

Arms and Interventions

Arm 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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Long Term Follow-Up on Survival [12/01/2019 - 12/31/2022]

      The primary outcome measure will be overall survival.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Cyprium Study Team New York New York United States 10014

    Sponsors and Collaborators

    • Cyprium Therapeutics, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Cyprium Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT04337684
    Other Study ID Numbers:
    • CYP-002
    First Posted:
    Apr 8, 2020
    Last Update Posted:
    Jul 18, 2022
    Last Verified:
    Jul 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Menkes Kinky Hair Syndrome

    Study Results

    No Results Posted as of Jul 18, 2022