Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously W… (NCT05739643) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
United States9 participantsStarted 2023-02-03
Plain-language summary
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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. 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:
✓. Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
✓. Psychosine levels predictive of infantile onset by Dried Blood Spot (DBS); OR
✓. Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
✓. Two GALC mutations predictive to result in infantile onset phenotype.
✓. 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:
✓. Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of late infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
✓. Psychosine levels predictive of late infantile onset by DBS; OR
Exclusion criteria
✕. 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.
What they're measuring
1
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101
✕. History of prior treatment with a gene therapy product
✕. Motor function evaluated by age with PDMS-II by a study physical therapist:
✕. 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.
✕. Grade 2 or higher abnormalities in LFTs, bilirubin, creatinine, white count, hemoglobin, platelets, PT/INR and PTT according to latest version of CTCAE
✕. Presence of any neurocognitive deficit, motor deficit, or brain damage not attributable to Krabbe disease
✕. 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.
✕. Active bacterial or fungal infection documented the preceding 7 days.