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Gene Therapy

Gene Therapy for Krabbe Disease (REKLAIM Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Forge Biologics, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months and 24 months
Awards & highlights

REKLAIM Trial Summary

This trial will study a gene therapy to treat Krabbe disease in people who have had a stem cell transplant. Data from untreated and already-transplanted patients will be compared.

Who is the study for?
This trial is for children with Krabbe Disease who've had a stem cell transplant at least 90 days before. They must have certain diagnostic criteria met, including specific test results or mutations, and sufficient engraftment of donor cells. Children can't join if they have recent infections, MRI or lumbar puncture contraindications, used investigational products recently, live virus immunizations within the last month, severe organ function issues, neurocognitive deficits not due to Krabbe disease, heart problems without pulmonary hypertension evidence or previous gene therapy treatments.Check my eligibility
What is being tested?
The study tests FBX-101 in kids who previously received a stem cell transplant for Krabbe Disease. It's an escalating dose trial where participants get one infusion of this gene therapy product. The effects will be compared to data from untreated and transplanted patients with infantile and late infantile forms of the disease.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to immune response against the viral vector (like fever), complications from underlying conditions exacerbated by treatment or typical risks associated with infusions such as discomfort at injection site.

REKLAIM Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 longitudinally by PDMS-2, BOTMP2, or by GMFM-88, depending on the age, compared to patients receiving HSCT only

REKLAIM Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention
N=3-6 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the high dose
Group II: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention
N=3 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the low dose

Find a Location

Who is running the clinical trial?

Forge Biologics, IncLead Sponsor
2 Previous Clinical Trials
31 Total Patients Enrolled

Media Library

FBX-101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05739643 — 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: NCT05739643 — Phase 1 & 2
FBX-101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05739643 — Phase 1 & 2
Krabbe Disease Patient Testimony for trial: Trial Name: NCT05739643 — Phase 1 & 2

Frequently Asked Questions

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

Are there any unfilled positions in this research trial?

"As per the recent updates on clinicaltrials.gov, this experiment is still open for recruitment. The trial was initially posted on February 3rd 2023 and has been since edited as of February 13th 2023."

Answered by AI

What is the limit for cohort size in this clinical trial?

"Affirmative. According to clinicaltrials.gov, the study is still recruiting patients; it was initially posted on February 3rd 2023 and last adjusted on February 13th 2023. For this trial, 12 individuals must be recruited from a single site."

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
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

Krabbe deseas treatment.
PatientReceived 1 prior treatment
Recent research and studies
clinicaltrials.govStudy
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
2023. "Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05739643.
All > Krabbe Disease
~6 spots leftby Jul 2026
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