Metachromatic Leukodystrophy (MLD) Clinical Trial
Official title:
Gene Therapy for Metachromatic Leukodystrophy (MLD) Using a Self-inactivating Lentiviral Vector (TYF-ARSA)
This is a Phase I/II clinical trial of gene transfer for treating Metachromatic leukodystrophy (MLD) using a safety and efficiency 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 gene transfer clinical protocol.
Status | Recruiting |
Enrollment | 10 |
Est. completion date | November 1, 2020 |
Est. primary completion date | October 30, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month and older |
Eligibility |
Inclusion Criteria: 1. MLD patient age >= 0 year 2. ARSA gene sequence analysis to confirm MLD mutations 3. Scoring system for brain MR Imaging confirmed MLD 4. Parent / guardian / patient signing informed consent 5. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form Exclusion Criteria: 1. HIV positive patients 2. Patients who are 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 pre-injection site 5. Any condition that may increase the subjects' risk or interfere with the results of the trial. In addition to MLD, there are other neurological disorders. |
Country | Name | City | State |
---|---|---|---|
China | Lung-Ji Chang | Shenzhen | Guangdong |
Lead Sponsor | Collaborator |
---|---|
Shenzhen Geno-Immune Medical Institute |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Safety of intracerebral injection of lentiviral TYF-ARSA. | Safety of intracerebral injection 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 study drug(s). Grade 1 & 2 AEs will be summarized if related to study therapy. | up to 1 year follow up | |
Primary | Altered disease progression | Altered disease progression based on biochemical analysis and MRI brain imaging analysis. | up to 3 year follow up after treatment |
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