Direct Lentiviral Injection 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 functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.
Eligibility criteria
Qualifiers
MLD patient age >= 1 month
ARSA gene sequence analysis to confirm MLD mutations
Scoring system for brain MR Imaging confirmed MLD
Parent / guardian / patient signing informed consent
Disqualifiers
HIV positive patients
Patients who are experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
Cannot perform an MRI
Infection or dermatosis at pre-injection site
Trial design
Treatments tested in this trial
- Intrathecal and intravenous LV gene therapy
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov