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Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
Sponsor: Duke University
Summary
This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.
Official title: A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
2
Start Date
2024-08-06
Completion Date
2025-04-11
Last Updated
2026-11-04
Healthy Volunteers
No
Conditions
Interventions
FBX-101
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (hGALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
Locations (1)
Duke University
Durham, North Carolina, United States