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NCT02714764 · ClinicalTrials.gov registry record
Evaluation of Outcome Metrics in Alexander Disease
A clinical trial of Alexander Disease, sponsored by Children's Hospital of Philadelphia.
- Recruiting
- Registry status
- 200
- Enrollment target
- 1
- Study location
NCT02714764: Recruiting study of Alexander Disease, sponsored by Children's Hospital of Philadelphia.
NCT02714764 is a study of Alexander Disease that is actively recruiting participants, run by Children's Hospital of Philadelphia. The registered enrollment target is 200 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02714764, a study of Alexander Disease, is actively recruiting participants, sponsored by Children's Hospital of Philadelphia.
- RECRUITING
- Registry status
- 200 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. Specimens are collected to measure glial fibrillary acidic protein (GFAP) levels in cerebrospinal fluid (CSF) and blood. The data obtained from this study will be used for the design of future treatment trials.
Primary Outcome
Total score and dimensional scores (rolling/supine, crawling/traveling, sitting, standing, and walking/running) will be calculated at each visit. Change in total and dimensional scores over time will be assessed.
Conditions Studied
Study Locations (1)
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 200 participants |
| Start Date | 2016-01-26 |
| Est. Completion | 2030-12 |
What NCT02714764 shows while recruiting
NCT02714764 is an observational study that tracks outcomes without assigning an intervention. The registered 200 participants enrollment target is mid-sized for trials with a published cap.
The record links to 1 condition, with Alexander Disease appearing as the primary indexed condition, and to 0 interventions.
NCT02714764 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT02714764 about?
NCT02714764 is a clinical study titled "Evaluation of Outcome Metrics in Alexander Disease". The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallow...
What is the current status of trial NCT02714764?
This trial is currently recruiting. The enrollment target is 200 participants. The study started on 2016-01-26. Estimated completion is 2030-12.
What conditions does trial NCT02714764 study?
This clinical trial studies the following conditions: Alexander Disease.
Who is sponsoring clinical trial NCT02714764?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02714764 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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