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NCT03333200 · ClinicalTrials.gov registry record
Longitudinal Study of Neurodegenerative Disorders
A clinical trial of Tay-Sachs Disease and Krabbe Disease, sponsored by University of Pittsburgh.
- Recruiting
- Registry status
- 1,500
- Enrollment target
- 1
- Study location
NCT03333200: Recruiting study of Tay-Sachs Disease and Krabbe Disease, sponsored by University of Pittsburgh.
NCT03333200 is a study of Tay-Sachs Disease and Krabbe Disease that is actively recruiting participants, run by University of Pittsburgh. The registered enrollment target is 1,500 participants, above the 77-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (1848% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03333200, a study of Tay-Sachs Disease and Krabbe Disease, is actively recruiting participants, sponsored by University of Pittsburgh.
- RECRUITING
- Registry status
- 1,500 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.
Primary Outcome
Repeated standardized age equivalent scores.
Conditions Studied
Interventions
- OTHER Palliative Care
- BIOLOGICAL Hematopoetic Stem Cell Transplantation
Study Locations (1)
Pennsylvania
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1,500 participants |
| Start Date | 2012-01-11 |
| Est. Completion | 2035-01 |
What NCT03333200 shows while recruiting
NCT03333200 is an observational study that tracks outcomes without assigning an intervention. Its 1,500 participants enrollment target places it among the larger protocols in the corpus, above the 77-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (1848% higher).
The record links to 10 conditions, with Tay-Sachs Disease appearing as the primary indexed condition, and to 2 interventions - of which Palliative Care is the first listed.
NCT03333200 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT03333200 about?
NCT03333200 is a clinical study titled "Longitudinal Study of Neurodegenerative Disorders". The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.
What is the current status of trial NCT03333200?
This trial is currently recruiting. The enrollment target is 1,500 participants. The study started on 2012-01-11. Estimated completion is 2035-01.
What conditions does trial NCT03333200 study?
This clinical trial studies the following conditions: Tay-Sachs Disease, Krabbe Disease, MPS I, Vanishing White Matter Disease, ALD.
What interventions are being tested in trial NCT03333200?
The interventions under investigation include: Palliative Care (OTHER), Hematopoetic Stem Cell Transplantation (BIOLOGICAL).
Who is sponsoring clinical trial NCT03333200?
This trial is sponsored by University of Pittsburgh, which has 1,238 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03333200 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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