Direct Lentiviral Injection Gene Therapy for MLD
ConditionMetachromatic Leukodystrophy (MLD)
Trial statusNot yet recruiting
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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
Intrathecal and intravenous LV gene therapy
GeneticDirect IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 transduction units/ml which carry a normal ARSA gene
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: Lentivirus-mediated delivery of ARSA to the CNS and the bodyExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety of IT and IV injections of lentiviral TYF-ARSA
Safety of IT and IV injections of 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 by maximum intensity \& relationship to the study drug(s). The grade of AEs will be summarized if related to the treatment.
Time frame
up to 1 year follow up2
Altered disease progression
Altered disease progression based on biochemical analysis.
Time frame
up to 3 year follow up after treatment3
Altered disease progression
Altered disease progression based on MRI brain imaging analysis
Time frame
up to 3 year follow up after treatmentSponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: