Krabbe Disease Treatment with FBX-101 Gene Transfer Therapy: The RESKUE Study – Phase I/II

Study of Intravenous Gene Transfer with an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation

Protocol Description

The purpose of this study is to assess the safety and potential effectiveness of using gene therapy for infants with Krabbe disease following hematopoietic stem cell transplantation (HSCT), the current standard of care. The drug FBX-101, also called AAVrh10, delivers a normal copy of the GALC gene to those cells in the child’s central and peripheral nervous systems that may not be addressed by transplant. The goal is to restore normal production of the galactocerebrosidase (GALC) enzyme, which is deficient in patients with Krabbe disease.

Study Level: Phase I/II

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling infants from birth through 12 months of age who have been diagnosed with Krabbe disease.
Males and Females: Birth through 12 months


Participants will be given chemotherapy about 10 days before HSCT, and a one-time dose of FBX-101 will be administered about 21 days later, including a 2-week hospital stay for monitoring. There will be 12 outpatient follow-up visits over the next 2 years to record progress of the disease and any side effects from treatment.
Visits: About 13, plus hospitalization for HSCT and gene therapy
Duration: About 26 months (2 years, 2 months)

Status: Closed to Enrollment

Source(s) of Support

Forge Biologics, Inc.

Primary Investigator

Randy Windreich, MD

Contact Information

For more information about the study or enrollment, please contact:
Brie Yanniello, NDRD Program Coordinator

*Original research initiated by investigators at UPMC Children’s Hospital of Pittsburgh.