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NCT00668187 · ClinicalTrials.gov registry record
A Natural History Study of the Gangliosidoses
A clinical trial of GM1 Gangliosidosis and Tay-Sachs Disease, sponsored by University of Minnesota.
- Recruiting
- Registry status
- 52
- Enrollment target
- 1
- Study location
NCT00668187: Recruiting study of GM1 Gangliosidosis and Tay-Sachs Disease, sponsored by University of Minnesota.
NCT00668187 is a study of GM1 Gangliosidosis and Tay-Sachs Disease that is actively recruiting participants, run by University of Minnesota. The registered enrollment target is 52 participants, below the 98-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (47% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00668187, a study of GM1 Gangliosidosis and Tay-Sachs Disease, is actively recruiting participants, sponsored by University of Minnesota.
- RECRUITING
- Registry status
- 52 participants
- Enrollment target
- 1
- Study location
Study Summary
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in order to better understand the natural history and heterogeneity of these diseases. Such a quantitative method will also be essential for evaluating any treatments that may become available in the future, such as gene therapy. The data from this study will be necessary to provide end-points for future therapies, guide medical decisions about treatment, provide objective measurement of treatment outcomes, and accurately inform parents regarding potential outcomes.
Primary Outcome
Neuropsychological testing data will be collected at baseline and annually, that measure fine and gross motor skills, visual tracking and attention, verbal and non-verbal communication, and emotional and social behaviors. For infantile and juvenile Tay-Sachs disease, Sandhoff disease and GM1 gangliosidosis-affected subjects, age- and ability-appropriate neurobehavioral and neurodevelopmental testing will include instruments such as the Bayley Scales of Infant Development (Third Edition), and the
Conditions Studied
Study Locations (1)
Minnesota
- University of Minnesota - Pediatric Genetics and Metabolism - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 52 participants |
| Start Date | 2010-12 |
| Est. Completion | 2027-03-01 |
What NCT00668187 shows while recruiting
NCT00668187 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 52 participants, a relatively small participant target, below the 98-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (47% lower).
The record links to 5 conditions, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 0 interventions.
NCT00668187 reports a single indexed study location in Minnesota.
Frequently Asked Questions
What is clinical trial NCT00668187 about?
NCT00668187 is a clinical study titled "A Natural History Study of the Gangliosidoses". Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in...
What is the current status of trial NCT00668187?
This trial is currently recruiting. The enrollment target is 52 participants. The study started on 2010-12. Estimated completion is 2027-03-01.
What conditions does trial NCT00668187 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease, GM2 Gangliosidosis, Late Onset Tay-Sachs Disease.
Who is sponsoring clinical trial NCT00668187?
This trial is sponsored by University of Minnesota, which has 966 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00668187 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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