NCT02714764 Evaluation of Outcome Metrics in Alexander Disease
| NCT ID | NCT02714764 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Alexander Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 200 participants |
| Start Date | 2016-01-26 |
| Primary Completion | 2030-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 200 participants in total. It began in 2016-01-26 with a primary completion date of 2030-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * Diagnosed with Alexander Disease Exclusion Criteria: * Other Leukodystrophies will not be enrolled
Contact & Investigator
Amy Waldman, MD
📞 215-590-1719
Amy Waldman, MD
PRINCIPAL INVESTIGATOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT02714764 clinical trial?
This trial is open to participants of all sexes, studying Alexander Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT02714764 currently recruiting?
Yes, NCT02714764 is actively recruiting participants. Visit ClinicalTrials.gov or contact Children's Hospital of Philadelphia to inquire about joining.
Where is the NCT02714764 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT02714764 clinical trial?
NCT02714764 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Amy Waldman, MD at Children's Hospital of Philadelphia. The trial plans to enroll 200 participants.