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Evaluation of Outcome Metrics in Alexander Disease
NCT02714764 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02714764
The ClinicalTrials.gov registry entry for NCT02714764 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 200 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital of Philadelphia, which has 492 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Alexander Disease appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02714764 reports 1 study location spanning 1 distinct geographic area — top geographies include Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT02714764?
This trial is sponsored by Children's Hospital of Philadelphia, which has 492 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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