What side effects are associated with this type of intervention?

The most common side effects of gene therapy treatments for Krabbe Disease, such as Adeno-Associated Virus (AAV) gene therapy, include immune responses to the viral vector, which can cause inflammation or other immune-mediated reactions. There is also a risk of insertional mutagenesis, potentially leading to malignancies. Other side effects may include mild to moderate symptoms like fever, headache, and fatigue.

What prior FDA approvals exist?

As of now, there are no FDA-approved gene therapies specifically for Krabbe Disease. Current treatments primarily focus on hematopoietic stem cell transplantation (HSCT), which can slow disease progression if administered early. Investigational therapies, including adeno-associated virus (AAV) gene therapy, are being studied to correct the genetic defect in Krabbe Disease, but these are still in clinical trial phases and have not yet received FDA approval.

What other types of research exist?

Promising novel alternatives to Adeno-Associated Virus (AAV) gene therapy for Krabbe Disease patients include: 1) Hematopoietic Stem Cell Transplantation (HSCT) combined with gene editing techniques such as CRISPR/Cas9 to correct genetic defects in stem cells before transplantation. 2) Enzyme Replacement Therapy (ERT) using recombinant human galactocerebrosidase to replace the deficient enzyme in patients. 3) Small molecule therapies that enhance the residual activity of the defective enzyme or reduce the accumulation of toxic substrates. These approaches are being actively researched and may offer effective treatment options in the near future.

← Back to Search

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

No Placebo-Only Group

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

Select...

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

Select...

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

Select...

My child has Krabbe disease with specific test results.

Select...

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:

Select...

I have been treated with gene therapy before.

Select...

I am not currently in another clinical trial or using any experimental treatments.

Select...

I have an ongoing and worsening bacterial or fungal infection.

Select...

I have a liver condition diagnosed by ultrasound.

Select...

I have a birth defect or condition that affects my brain development.

Select...

I do not have any signs of active infection or disease from specific viruses.

Select...

I have brain issues not caused by Krabbe disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months and 24 months

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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)

0 / 11Eligibility criteria met

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