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Gene Therapy

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

Phase 1 & 2
Waitlist Available
Led By Jessie Barnum, MD
Research Sponsored by Forge Biologics, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age at the time of screening: 1 day to 12 months
Participant must have adequate organ function at time of screening as measured by specific criteria for creatinine, hepatic transaminases, ejection fraction, pulmonary evaluation, and coagulation tests
Must not have
History of prior treatment with a gene therapy product
Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months and 24 months
Awards & highlights

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects may include reactions related to the infusion of FBX-101 or complications from the underlying condition and transplantation process; however, specific side effects are not detailed here.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a baby aged between 1 day and 12 months.
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My kidneys, liver, heart, lungs, and blood clotting functions are all within normal ranges.
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My child has Krabbe disease with specific test results.
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I am eligible for a stem cell transplant with a specific prep treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been treated with gene therapy before.
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I am not currently in another clinical trial or using any experimental treatments.
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I have an ongoing and worsening bacterial or fungal infection.
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I have a liver condition diagnosed by ultrasound.
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I have a birth defect or condition that affects my brain development.
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I do not have any signs of active infection or disease from specific viruses.
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I have brain issues not caused by Krabbe disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months and 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Other study objectives
Exploratory endpoint as assessed by change in auditory brainstem responses compared to patients receiving HSCT only.
Exploratory endpoint as assessed by change in brain MRI as measured by DTI compared to patients receiving HSCT only.
Body Weight Changes
+22 more

Trial Design

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)

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatment for Krabbe Disease being studied is gene therapy using adeno-associated virus (AAV) vectors. This approach involves delivering a functional copy of the defective gene into the patient's cells. The AAV vector is engineered to carry the healthy gene and, once administered, it targets the affected cells, allowing them to produce the necessary enzyme that is deficient in Krabbe Disease. This is crucial for patients because it addresses the root cause of the disease at a genetic level, potentially halting or reversing the progression of the disease, which is otherwise fatal and currently has limited treatment options.

Find a Location

Who is running the clinical trial?

Forge Biologics, IncLead Sponsor
2 Previous Clinical Trials
34 Total Patients Enrolled
Jessie Barnum, MDPrincipal InvestigatorDiv. of Blood & Marrow Transplantation & Cellular Therapies, UPMC Children's Hospital of Pittsburgh
1 Previous Clinical Trials
25 Total Patients Enrolled
Randy WindreichPrincipal InvestigatorClinical Director, Division of Blood and Marrow Transplantation and Cellular Therapies, UPMC Children's Hospital of Pittsburgh

Media Library

FBX-101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04693598 — Phase 1 & 2
Krabbe Disease Research Study Groups: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC), Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)
Krabbe Disease Clinical Trial 2023: FBX-101 Highlights & Side Effects. Trial Name: NCT04693598 — Phase 1 & 2
FBX-101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04693598 — Phase 1 & 2
~2 spots leftby Oct 2025