Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
Recruiting
N/A
Interventional Study
Metachromatic Leukodystrophy (MLD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 1 Month – 50
- Sex
- Any
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Shenzhen Geno-Immune Medical Institute · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
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 safe…
Trial Locations
Loading…
Loading trial locations…
Eligibility 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 conseque…
Contacts