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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT03725670 |
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Date of registration:
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25/09/2018 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Lentiviral Gene Therapy for MLD
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Scientific title:
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Gene Therapy for Metachromatic Leukodystrophy (MLD) Using a Self-inactivating Lentiviral Vector (TYF-ARSA) |
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Date of first enrolment:
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October 30, 2018 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03725670 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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N/A
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Countries of recruitment
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China
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Contacts
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Name:
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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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86-0755-86725195 |
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Email:
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c@szgimi.org |
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Affiliation:
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Name:
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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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86-0755-86725195 |
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Email:
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c@szgimi.org |
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Affiliation:
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Name:
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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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Email:
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Affiliation:
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Shenzhen Geno-Immune Medical Institute |
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Key inclusion & exclusion criteria
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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.
Age minimum:
1 Month
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Metachromatic Leukodystrophy (MLD)
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Intervention(s)
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Biological: Lentivirus-mediated delivery of ARSA to the CNS.
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Primary Outcome(s)
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Altered disease progression
[Time Frame: up to 3 year follow up after treatment]
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Safety of intracerebral injection of lentiviral TYF-ARSA.
[Time Frame: up to 1 year follow up]
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Secondary ID(s)
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GIMI-IRB-18005
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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