Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT (REKLAIM)

November 19, 2025 updated by: Forge Biologics, Inc

A Phase 1/2 Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)

This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The FBX-101-REKLAIM study has been modified on Q4 2023 to allow a broader patient recruitment of infantile and late infantile Krabbe patients. The updated REKLAIM study merges the recruitment populations of the previous FBX-101-RESKUE clinical trial (NCT04693598) and the FBX-101-REKLAIM clinical trial (NCT05739643).

Study Type

Interventional

Enrollment (Estimated)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Orange, California, United States, 92868
        • Children's Hospital of Orange County (CHOC)
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Hospitals - Michigan Medicine
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Group Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for asymptomatic infantile onset Krabbe disease with initial diagnosis based on:

    1. Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
    2. Psychosine levels predictive of infantile onset by Dried Blood Spot (DBS); OR
    3. Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
    4. Two GALC mutations predictive to result in infantile onset phenotype.

  2. Group Late Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for symptomatic late infantile onset Krabbe with initial diagnosis based on:

    1. Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of late infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
    2. Psychosine levels predictive of late infantile onset by DBS; OR
    3. Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
    4. Two GALC mutations predictive to result in late infantile onset phenotype; OR
    5. Neurological/developmental exam findings consistent with late infantile Krabbe disease
  3. Participants must be considered candidates for HSCT or have received HSCT at least 21 days prior to dosing date
  4. For patients already transplanted and followed for more than 3 months chimerism should reflect at least 30% of myeloid cells from the donor by month 3 post-transplant, from 30 to 10% between 3 months and one year post-transplant or 10% by one year post-transplant.

  5. Participant must have adequate organ function at time of screening or evaluation as measured by:

    1. Ejection fraction of > 50% by echocardiogram or other appropriate study without evidence of pulmonary hypertension.
    2. Pulmonary evaluation testing demonstrating resting pulse oximeter > 95% on room air.
  6. Absence of active aspiration
  7. Participant's parents or legal guardian consent to participate in the study and provide informed consent according to IRB/IEC guidelines prior to any study procedures being performed
  8. Parent(s) and/or legal guardian able to comply with the clinical protocol

Exclusion Criteria:

  1. Immunoassay with total anti-AAV10 antibody titers of >1:100. This criterion will not apply to children screened before they have received HSCT or for children who sign the inform consent within 6 months from HSCT. In children who test positive to anti-AAV10 antibody with titers of >1:100 under this exception, the ISR regime proposed by the PI and approved/modified by the ISR committee may include immunosuppressive drugs that prevent the potential development of a secondary immune response to AAVrh10 after FBX-101 administration.
  2. History of prior treatment with a gene therapy product

  3. Motor function evaluated by age with PDMS-II by a study physical therapist:

    a. Inability to hold head for patients older than 5 months; b. Inability to sit independently for patients older than 12 months; c. Inability to walk with assistance for patients older than 24 months.

  4. In patients that sign the informed consent before HSCT or up to 90 days post-HSCT, abnormalities in white count, hemoglobin and platelets found from conditioning regime to Day -1 (the day before FBX-101 administration) will be evaluated by the PI (with referral to the DSMB if indicated). If abnormal, they will not be considered an exclusion criteria if the PI considers they are consistent with expected consequences of the HSCT (and related management) and upon confirmation they were not present before commencement of the conditioning regime.
  5. Grade 2 or higher abnormalities in LFTs, bilirubin, creatinine, white count, hemoglobin, platelets, PT/INR and PTT according to latest version of CTCAE
  6. Presence of any neurocognitive deficit, motor deficit, or brain damage not attributable to Krabbe disease
  7. Signs of active infections or disease from cytomegalovirus, adenovirus, EBV, hepatitis B or C, and HIV or other viruses excluding rhinovirus from RVP and asymptomatic norovirus presence in stool. Patients showing HIV positive results will be excluded from the study.
  8. Active bacterial or fungal infection documented the preceding 7 days.
  9. Presence of any contraindication for MRI or lumbar puncture (LP)
  10. Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions
  11. Immunizations with live viruses in the 30 days prior to immune suppression
  12. Active acute Graft Versus Host Disease (GvHD) Grade II or higher according to modified Glucksberg criteria (Przepiorka et al., 1995) or active, moderate or severe, chronic GvHD according to revised NIH criteria (Jagasia et al., 2015)
  13. Any other medical condition, serious intercurrent illness, other genetic condition or extenuating circumstance that, in the opinion of the PI, would preclude participation in the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)
N=3 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the low dose
Biological: FBX-101
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
Other Names:
  • AAVrh.10-hGALC
Experimental: Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)
N=3-6 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the high dose
Biological: FBX-101
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
Other Names:
  • AAVrh.10-hGALC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101
Time Frame: 24 months
24 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Efficacy as assessed by improvement of gross motor function as measured longitudinally by PDMS-2, BOT-3, or by GMFM-88, depending on the age, compared to patients receiving HSCT only
Time Frame: 12 months and 24 months
12 months and 24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Forge Biologics, Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 3, 2023

Primary Completion (Estimated)

November 1, 2026

Study Completion (Estimated)

November 1, 2026

Study Registration Dates

First Submitted

February 13, 2023

First Submitted That Met QC Criteria

February 13, 2023

First Posted (Actual)

February 22, 2023

Study Record Updates

Last Update Posted (Actual)

November 20, 2025

Last Update Submitted That Met QC Criteria

November 19, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FBX-101-REKLAIM

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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