Lentiviral gene therapy using a patient's own blood-forming stem cells for MLD
Lentiviral Vector-modified Autologous Hematopoietic Stem Cells for Metachromatic Leukodystrophy (MLD)
This treatment will try to fix a patient's own hematopoietic stem cells with a lentiviral ARSA gene to treat people with metachromatic leukodystrophy.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 1 Month to 50 Years |
| Sex | All |
| Sponsor | Shenzhen Geno-Immune Medical Institute Academic / other |
| Locations | 1 site (Shenzhen, Guangdong) |
| Trial ID | NCT07046338 on ClinicalTrials.gov |
What this trial studies
This Phase I/II program uses an improved self-inactivating lentiviral vector (TYF-ARSA) to insert a functional ARSA gene into a patient's harvested hematopoietic stem cells, which are then returned to the patient by transplant. The approach aims to restore ARSA enzyme activity in the nervous system by long-lived, gene-corrected blood cells and their progeny. Primary workstreams include monitoring safety (including vector integration sites) and measuring clinical and biochemical signs of disease stabilization or improvement. Genetic confirmation of ARSA mutations, brain MRI, and capacity to undergo stem cell collection and transplantation are required for enrollment, with long-term follow-up planned at the treating center in Shenzhen.
Who should consider this trial
Good fit: Ideal candidates are people of any age (≥1 month) with genetically confirmed ARSA mutations who can undergo hematopoietic stem cell collection and transplantation and who do not have uncontrolled infections or excluded comorbidities.
Not a fit: Patients with advanced, irreversible neurological damage, active uncontrolled serious infections, HIV positivity, or other conditions that prevent safe transplant are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this therapy could provide durable ARSA enzyme production from the patient's own cells and slow or halt the demyelination that causes MLD symptoms.
How similar studies have performed: Autologous lentiviral hematopoietic stem cell gene therapies for MLD have shown clinical benefit, particularly when given before or very early in symptoms, and similar approaches have received regulatory approval in some regions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Shenzhen, Guangdong
- Shenzhen Geno-Immune Medical Institute — Shenzhen, Guangdong, China (Recruiting)
Study contacts
- Study coordinator: Lung-Ji Chang, Ph.D
- Email: c@szgimi.org
- Phone: 86-13671121909
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.