Natural History Study of Patients With Canavan Disease (CANinform Study)

Last updated: January 9, 2025
Sponsor: Aspa Therapeutics
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

N/A

Clinical Study ID

NCT04126005
CVN-101
CANinform
  • All Genders

Study Summary

This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in children.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Meet age criteria of a specific cohort.

  2. Confirmed clinical and biochemical diagnosis of Canavan disease.

  3. Available medical records since birth that permit documentation of diseasecharacteristics and developmental milestones.

  4. Parent and/or legal guardian is able to read, understand, and sign the informedconsent.

Exclusion

Exclusion Criteria:

  1. Patient does not meet the Inclusion Criteria.

Study Design

Total Participants: 70
Study Start date:
October 10, 2019
Estimated Completion Date:
April 30, 2026

Study Description

The CANinform natural history study is the first multinational effort to rigorously gather both retrospective and prospective data from this patient population. Data collection includes extraction of retrospective data from medical records of living and deceased patients, and collection of prospective, longitudinal data from living patients and their parent(s)/caregiver(s). Motor function assessments are performed remotely in the home via video or in the clinic by qualified study team members. Families will be invited to attend clinic visits and/or will be followed remotely by the clinical site for approximately 3 years.

Connect with a study center

  • University Medical Center Hamburg-Eppendorf

    Hamburg, 20246
    Germany

    Active - Recruiting

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California 94609
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • NYU Langone Medical Center

    New York, New York 10016
    United States

    Site Not Available

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