Evaluation of Outcome Metrics in Alexander Disease
Launched by CHILDREN'S HOSPITAL OF PHILADELPHIA · Mar 16, 2016
Trial Information
Current as of June 06, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is studying Alexander Disease, a condition that affects the brain and can lead to problems with movement, speech, and overall quality of life. The researchers want to understand how this disease progresses over time and its impact on daily activities like walking, talking, and swallowing. To do this, they will collect information from participants and also gather samples from their blood and spinal fluid to measure certain proteins that may be related to the disease. The findings from this trial will help shape future treatment options for those affected by Alexander Disease.
To participate in this study, individuals must have a confirmed diagnosis of Alexander Disease. Unfortunately, those with other similar conditions, known as leukodystrophies, will not be able to join. The trial is currently looking for participants of all ages and genders. If eligible, you can expect to undergo assessments that will help the researchers understand the effects of Alexander Disease better. This research is crucial for developing new therapies and improving care for patients in the future.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Diagnosed with Alexander Disease
- Exclusion Criteria:
- • Other Leukodystrophies will not be enrolled
About Children's Hospital Of Philadelphia
The Children's Hospital of Philadelphia (CHOP) is a leading pediatric healthcare institution renowned for its commitment to advancing child health through innovative research and clinical care. As a prominent clinical trial sponsor, CHOP emphasizes a multidisciplinary approach to pediatric research, fostering collaborations across various specialties to develop and evaluate groundbreaking therapies and interventions. With a focus on improving patient outcomes and enhancing the quality of life for children, CHOP is dedicated to conducting rigorous clinical trials that adhere to the highest ethical standards and scientific integrity, ultimately contributing to the global knowledge base in pediatric medicine.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Philadelphia, Pennsylvania, United States
Patients applied
Trial Officials
Amy Waldman, MD
Principal Investigator
Children's Hospital of Philadelphia
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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