A Natural History Study of Canavan Disease
1 other identifier
observational
21
2 countries
3
Brief Summary
The purpose of this study is to learn more about the natural history of Canavan disease
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started May 2016
Typical duration for all trials
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2016
CompletedFirst Submitted
Initial submission to the registry
July 28, 2016
CompletedFirst Posted
Study publicly available on registry
August 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2019
CompletedNovember 1, 2019
October 1, 2019
2.9 years
July 28, 2016
October 31, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Change in Gross Motor Function Measure over time
baseline, 6 months, 1 year, 18 months, 2 years
Secondary Outcomes (1)
Change in magnetic resonance imaging findings over time
baseline, 6 months, 1 year, 18 months, 2 years
Eligibility Criteria
Subjects Diagnosed with Canavan Disease
You may qualify if:
- The patient must have a confirmed diagnosis of Canavan Disease as defined by elevated NAA levels, decreased ASPA activity, or mutations in the ASPA gene.
You may not qualify if:
- The PI will assess whether it is in the best interest of the patient to exclude them from the study for their own comfort and well being. In cases where the PI deems it appropriate, severely affected patients will be excluded
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- NYU Langone Healthlead
- Universitätsklinikum Hamburg-Eppendorfcollaborator
Study Sites (3)
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
NYU Langone Medical Center
New York, New York, 10016, United States
University Medical Center Hamburg-Eppendorf
Hamburg, 20246, Germany
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Florian Eichler, MD
Massachusetts General Hospital
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 28, 2016
First Posted
August 1, 2016
Study Start
May 1, 2016
Primary Completion
April 1, 2019
Study Completion
April 1, 2019
Last Updated
November 1, 2019
Record last verified: 2019-10
Data Sharing
- IPD Sharing
- Will share
Data from this study will be stored in a computer data repository at the Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI). The purpose of this data repository is to capture and store data for clinical research. The repository will combine data from multiple studies. Datasets will be shared with researchers who want to advance understanding of Neurological Disease. These datasets will not contain any personal identifiable information.