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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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months and 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months and 24 months
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I had a stem cell transplant more than 90 days ago.I am getting or have had a transplant for Krabbe disease diagnosed in infancy.Your parents or legal guardian give permission for you to participate in the study and understand the information provided by the study organizers.I have not received any live virus vaccines in the last 30 days.I have a moderate to severe reaction from a transplant.I cannot lift my arms against gravity.I have brain or nerve issues not caused by Krabbe disease.My bone marrow transplant is showing signs of taking hold.Your parent(s) or legal guardian can follow the study guidelines.I have been treated with gene therapy before.I had a transplant over 3 months ago and meet the chimerism requirements.I do not have any signs of active infections or diseases from viruses.I have had a bacterial or fungal infection in the last week.I am not currently in another clinical study or using any experimental treatments.I am eligible for a stem cell transplant or had one at least 21 days ago.I am being or have been transplanted for late infantile Krabbe disease.My organs are functioning well according to tests.My tests show signs of Krabbe disease or I have two GALC mutations.I have high psychosine levels or tests showing Krabbe disease.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)
- Group 2: Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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
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