This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.
age >= 1 month
ARSA gene sequence analysis to confirm MLD mutations
Brain MR Imaging
Parent / guardian / patient signing informed consent
HIV positive
Experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
Cannot perform an MRI
Infection or dermatosis at infusion site
Single group
Lentiviral TYF-ARSA correction of patient's autologous HSCs
GeneticInfusion of lentiviral TYF-ARSA modified autologous HSCs at 1\~10x10\^6 gene-modified cells per kg body weight.
Safety of auto-HSCs transduced with lentiviral TYF-ARSA
Safety of HSCs transduced with lentiviral TYF-ARSA, determined by number of participants with treatment-related adverse events (AEs), according to scheduled assessments, vital signs, \& physical examinations as assessed by CTCAE v4.0. AEs \& clinically significant abnormalities (meeting grade 3, 4, or 5 criteria according to CTCAE) will be summarized. AEs will be graded if related to the treatment.
Altered disease progression
Altered disease progression based on biochemical analysis.
Altered disease progression
Altered disease progression based on MRI brain imaging analysis.
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This trial is not recruiting at the moment. You can still explore other options:
