Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
NCT ID: NCT05739643
Last Updated: 2025-11-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2023-02-03
2026-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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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
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.
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
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.
Interventions
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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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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.
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
18 Years
ALL
No
Sponsors
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Forge Biologics, Inc
INDUSTRY
Responsible Party
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Locations
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Children's Hospital of Orange County (CHOC)
Orange, California, United States
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
Countries
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References
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Wright MD, Poe MD, DeRenzo A, Haldal S, Escolar ML. Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology. 2017 Sep 26;89(13):1365-1372. doi: 10.1212/WNL.0000000000004418. Epub 2017 Aug 30.
Gupta A, Poe MD, Styner MA, Panigrahy A, Escolar ML. Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease. Neuroimage Clin. 2014 Sep 26;7:792-8. doi: 10.1016/j.nicl.2014.09.014. eCollection 2015.
Bascou N, DeRenzo A, Poe MD, Escolar ML. A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life. Orphanet J Rare Dis. 2018 Aug 9;13(1):126. doi: 10.1186/s13023-018-0872-9.
Beltran-Quintero ML, Bascou NA, Poe MD, Wenger DA, Saavedra-Matiz CA, Nichols MJ, Escolar ML. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis. 2019 Feb 18;14(1):46. doi: 10.1186/s13023-019-1018-4.
Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.
Escolar ML, West T, Dallavecchia A, Poe MD, LaPoint K. Clinical management of Krabbe disease. J Neurosci Res. 2016 Nov;94(11):1118-25. doi: 10.1002/jnr.23891.
Rafi MA, Luzi P, Wenger DA. Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease. Bioimpacts. 2020;10(2):105-115. doi: 10.34172/bi.2020.13. Epub 2020 Mar 24.
Yoon IC, Bascou NA, Poe MD, Szabolcs P, Escolar ML. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood. 2021 Apr 1;137(13):1719-1730. doi: 10.1182/blood.2020005477.
Escolar ML, Poe MD, Smith JK, Gilmore JH, Kurtzberg J, Lin W, Styner M. Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease. AJNR Am J Neuroradiol. 2009 May;30(5):1017-21. doi: 10.3174/ajnr.A1476. Epub 2009 Apr 22.
Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M. Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
Greco MR, Lopez MA, Beltran-Quintero ML, Tuc Bengur E, Poe MD, Escolar ML. Infantile Krabbe disease (0-12 months), progression, and recommended endpoints for clinical trials. Ann Clin Transl Neurol. 2024 Dec;11(12):3064-3080. doi: 10.1002/acn3.52114. Epub 2024 Nov 5.
Other Identifiers
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FBX-101-REKLAIM
Identifier Type: -
Identifier Source: org_study_id
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