Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03300453|
Recruitment Status : Completed
First Posted : October 3, 2017
Last Update Posted : December 2, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sanfilippo Syndrome B||Drug: rAAV2/5-hNAGLU||Phase 1 Phase 2|
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
Four patients, 18 months up to the 5th birthday, have been included.
The inclusion period will be 8 to 12 months. The duration of follow-up for each patient is 1 year post-surgery. The duration of the first extension phase is 18 months. The duration of the second extension phase is 36 months. Therefore, the maximum time of the follow-up will be 66 months
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-associated Viral Vector Containing the Human Alpha-N-acetylglucosaminidase cDNA in Children With Sanfilippo Type B Syndrome|
|Actual Study Start Date :||September 17, 2013|
|Actual Primary Completion Date :||November 27, 2019|
|Actual Study Completion Date :||November 27, 2019|
Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total).
one-time brain intraparenchymal gene therapy dose
Other Name: AAV5
- Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline [ Time Frame: Baseline until end of study (Month 66) ]Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.
- Number of Participants with presence of brain atrophy, white matter lesions and other lesions as assessed by cerebral MRI [ Time Frame: Baseline until end of study (Month 66) ]
MRIs at Baseline, Month 3, Month 12, Month 30, Month 48 and last visit Month 66.
Cerebral MRI will be collected for safety assessment to retrospectively evaluate for efficacy at Baseline, D0, Month 3, Month12, Month 30 and last visit Month 66.
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): NCT03300453
|Hopitaux Universitaires Paris-Sud|
|Paris, Le Kremlin-Bicetre Cedex, France, 94275|
|Principal Investigator:||Kumaran Deiva, MD||Hopitaux Universitaires Paris-Sud|