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New treatment option for Metachromatic Leukodystrophy (MLD)
Official title Direct Lentiviral Injection Gene Therapy for MLD
ClinicalTrials.gov ID: NCT03725670
What this study is testing
What is Intrathecal and intravenous LV gene therapy?
Intrathecal and intravenous LV gene therapy is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for metachromatic leukodystrophy (mld).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 50
You may be able to join if
- 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
- Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the...
You likely can't join if
- HIV positive patients
- Patients who are experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal...
- Cannot perform an MRI
- Infection or dermatosis at pre-injection site
- Any condition that may increase the people' risk or interfere with the results of the trial. In addition to MLD, there are other neurological...
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 month to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.