Gene Therapy with FBX-101 for Krabbe Disease
(RESKUE Trial)

Recruiting in Palo Alto (17 mi)

+2 other locations

Overseen byJessie Barnum, MD

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Forge Biologics, Inc

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?This trial tests a gene therapy given through an IV after a stem cell transplant in patients with Krabbe disease. The therapy uses a virus to deliver healthy genes to help fix the genetic problem. Gene therapy using viral vectors has shown some success in extending survival in mouse models of Krabbe disease.

Eligibility Criteria

This trial is for infants up to 12 months old diagnosed with infantile Krabbe disease, who are eligible for a stem cell transplant. They must have specific enzyme activity levels and organ function. Infants cannot participate if they have untreated infections, HIV, prior gene therapy, major congenital anomalies affecting brain development, or are in another clinical study.

Inclusion Criteria

I am a baby aged between 1 day and 12 months.

My kidneys, liver, heart, lungs, and blood clotting functions are all within normal ranges.

My child has Krabbe disease with specific test results.

+3 more

Exclusion Criteria

I have been treated with gene therapy before.

You are currently experiencing food or liquid going into your lungs.

You have HIV.

+8 more

Participant Groups

The trial tests FBX-101 after a standard stem cell transplant in babies with Krabbe disease. It's an escalating dose study of intravenous AAVrh10 gene therapy following the transplant. The effects will be compared to those observed in extensive natural history control groups.

2Treatment groups

Experimental Treatment

Group I: Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention

Participants will receive a single infusion at the higher dose (N=3 participants)

Group II: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention

Participants will receive a single infusion at the lower dose (N=3 participants)

FBX-101 is already approved in European Union, United States for the following indications:

🇪🇺 Approved in European Union as FBX-101 for: