Lentiviral Gene Therapy for MLD
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|ClinicalTrials.gov Identifier: NCT03725670|
Recruitment Status : Unknown
Verified September 2019 by Shenzhen Geno-Immune Medical Institute.
Recruitment status was: Recruiting
First Posted : October 31, 2018
Last Update Posted : September 19, 2019
|Condition or disease||Intervention/treatment||Phase|
|Metachromatic Leukodystrophy (MLD)||Biological: Lentivirus-mediated delivery of ARSA to the CNS.||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Gene Therapy for Metachromatic Leukodystrophy (MLD) Using a Self-inactivating Lentiviral Vector (TYF-ARSA)|
|Actual Study Start Date :||October 30, 2018|
|Actual Primary Completion Date :||October 30, 2018|
|Estimated Study Completion Date :||November 1, 2020|
Experimental: Lentivirus-mediated delivery of ARSA to the CNS.
Intracerebral injection with lentiviral TYF-ARSA vector carrying the functional gene
Biological: Lentivirus-mediated delivery of ARSA to the CNS.
Intracerebral LV gene therapy to deliver high level lenviral vectors which carry normal ARSA gene at 1-2×10^9 multiplicity of infection (moi)/ml per site.
- Safety of intracerebral injection of lentiviral TYF-ARSA. [ Time Frame: up to 1 year follow up ]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.
- Altered disease progression [ Time Frame: up to 3 year follow up after treatment ]Altered disease progression based on biochemical analysis and MRI brain imaging analysis.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03725670
|Contact: Lung-Ji Chang, Ph.Demail@example.com|
|Shenzhen, Guangdong, China, 518000|
|Contact: Lung-Ji Chang, Ph.D 86-0755-86725195 firstname.lastname@example.org|
|Principal Investigator:||Lung-Ji Chang, Ph.D||Shenzhen Geno-Immune Medical Institute|