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

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