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Clinical Trial Summary

This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04693598
Study type Interventional
Source Forge Biologics, Inc
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date November 5, 2021
Completion date July 2024

See also
  Status Clinical Trial Phase
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Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Terminated NCT00668564 - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Phase 2
Not yet recruiting NCT06308718 - Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Completed NCT01938014 - Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Recruiting NCT03333200 - Longitudinal Study of Neurodegenerative Disorders
Recruiting NCT05739643 - Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT Phase 1/Phase 2
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project
Completed NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Phase 2
Withdrawn NCT01425489 - Biomarker for Krabbe Disease (BioKrabbe)
Active, not recruiting NCT00787865 - Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease