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Gene Transfer Clinical Trial for Krabbe Disease

NCT04693598 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-01-30

No results posted yet for this study

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.

Conditions

  • Krabbe Disease

Interventions

BIOLOGICAL

FBX-101

A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.

Sponsors & Collaborators

  • Forge Biologics, Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Day
Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-05
Primary Completion
2026-11-30
Completion
2026-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04693598 on ClinicalTrials.gov