Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
ConditionMetachromatic Leukodystrophy (MLD)
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age1-50
SponsorShenzhen Geno-Immune Medical Institute
About this trial
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.
Eligibility criteria
Qualifiers
age >= 1 month
ARSA gene sequence analysis to confirm MLD mutations
Brain MR Imaging
Parent / guardian / patient signing informed consent
Disqualifiers
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
Trial design
Treatments tested in this trial
- Lentiviral TYF-ARSA correction of patient's autologous HSCs
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov