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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months and 24 months

Awards & highlights

RESKUE Trial Summary

This trial will test a new gene therapy for Krabbe disease. People with Krabbe disease will receive standard care for their disease, followed by a single infusion of the gene therapy. There will be a control group of people who have Krabbe disease who will not receive the gene therapy.

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.Check my eligibility

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.See study design

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.

RESKUE Trial 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.

RESKUE Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Safety as assessed by HSCT incident of engraftment.

Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101.

Secondary outcome measures

Efficacy as assessed by improvement of gross motor function as measured by Peabody Developmental Motor Scale 2nd Edition (PDMS-2) above a functional age equivalent of 12 months compared to untreated patients or those receiving HSCT only

Efficacy as assessed by improvement of probability to achieve independent sitting compared to untreated patients or those receiving HSCT only.

Other outcome measures

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

RESKUE 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 / 4Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can patients still join this trial?

"Yes, this clinical trial is recruiting patients right now. The original posting was on November 5th 2021, and the listing was updated as recently as August 24th 2022."

Answered by AI

How many guinea pigs are part of this experiment?

"That is accurate. The information available on clinicaltrials.gov suggests that the trial is still open and looking for participants. This particular study was posted on November 5th, 2021 and was most recently updated on August 24th, 2022. They are recruiting 6 more individuals at 2 sites."

Answered by AI

Might I be eligible to serve as a subject in this experiment?

"Researchers conducting this medical trial are looking for 6 individuals that suffer from leukodystrophy, globoid cell. In order to be eligible for the study, patients must meet the following age, disease, and health criteria: 1 day to 12 months old, predictive of infantile disease ≥ 9.0 nmol/L; OR Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR Two predicted pathogenic GALC mutations., Participant has been deemed eligible for treatment with HSCT (standard of care) or is within two weeks of HSC infusion, Participant"

Answered by AI

Will this clinical trial be testing the effects of the medication on patients over 55 years old?

"This study includes infants that are a day old up to toddlers that are 12 months old."

Answered by AI

Who else is applying?

What state do they live in?

Alabama

What site did they apply to?

University of Michigan Hospitals - Michigan Medicine

Children's Hospital of Orange County (CHOC)

UPMC Children's Hospital of Pittsburgh

What portion of applicants met pre-screening criteria?

Did not meet criteria

How many prior treatments have patients received?

Why did patients apply to this trial?

I am writing here because I heard you have some good results with fighting this disease. Noah is 11 month old, he has been recently diagnosed with Krabbe diseases – gallac gen. Noah is very flabby and stopped reaching for any toys, he has also never raise his head up. He also had actual umbilical cord knot, he was called a miracle baby because he was not supposed to survive because his mother had her uterus reconstructed and it was a miracle that she got pregnant. His mother is Polish and his father is from Salvador. Maybe you could help us to make another miracle? What are the options? What can we do here? After diagnose here in Poland we were told that there is nothing that can be done because he is already 11 months and has first symptom however we believe he is strong. He is eating on his own and is an adorable child. Please find the latest medical report (from hospital in Poland) attached. We are ocated in Poland but we can travel to USA.

PatientReceived no prior treatments

Recent research and studies

clinicaltrials.govStudy

Gene Transfer Clinical Trial for Krabbe Disease

2021. "Gene Transfer Clinical Trial for Krabbe Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04693598.

All > Krabbe Disease