Lentiviral TYF-ARSA correction of patient's autologous HSCs
Lentiviral TYF-ARSA correction of patient's autologous HSCs: Infusion of lentiviral TYF-ARSA modified autologous HSCs at 1\~10x10\^6 gene-modified cells per kg body weight.
Study summary
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
Sex
ALL
Min age
1 Month
Max age
50 Years
Healthy volunteers
No
Inclusion Criteria:
1. age \>= 1 month
2. ARSA gene sequence analysis to confirm MLD mutations
3. Brain MR Imaging
4. Parent / guardian / patient signing informed consent
5. Patients and their families have a strong willingness to participate in clinical trials, are willing to bear all the consequences caused by the failure of the trial, and sign the informed consent
Exclusion Criteria:
1. HIV positive
2. Experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
3. Cannot perform an MRI
4. Infection or dermatosis at infusion site
5. Any condition that may increase the subject's risk or interfere with the results of the trial, e.g. in addition to MLD, there are other neurological disorders.
Primary outcome measure(s)
Safety of auto-HSCs transduced with lentiviral TYF-ARSA — up to 1 year follow up 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 — up to 3 year follow up after treatment] Altered disease progression based on biochemical analysis.
Altered disease progression — up to 3 year follow up after treatment] Altered disease progression based on MRI brain imaging analysis.
Trial sites (1)
Facility
City
Region
Status
Shenzhen Geno-Immune Medical Institute
Shenzhen
Guangdong
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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