Indication
Krabbe Disease
6 clinical trials
4 products
Clinical trial
A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)Status: , Estimated PCD: 2027-01-01
Product
PBKR03Product
FBX-101Clinical trial
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)Status: Not yet recruiting, Estimated PCD: 2029-12-01
Clinical trial
A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)Status: Recruiting, Estimated PCD: 2026-07-01
Clinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01
Clinical trial
A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE)Status: Active (not recruiting), Estimated PCD: 2024-07-01
Clinical trial
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsStatus: Recruiting, Estimated PCD: 2024-10-01
Product
DUOC-01