A Phase 1/2 interventional study of rAAV2/5-hNAGLU in Sanfilippo Syndrome B, sponsored by UniQure Biopharma B.V.. Completed at 1 site in France. Open to participants aged 18 Months to 60 Months. Per ClinicalTrials.gov, last updated 2019-12-02.
Sponsored by UniQure Biopharma B.V. · Phase 1/2, Interventional, and Treatment
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.
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
55 studies on the registry are indexed under Mucopolysaccharidosis III; 16 are open to participants now.
This study's enrollment of 4 is below the median of 12 across 31 interventional studies indexed under Mucopolysaccharidosis III.
Browse Mucopolysaccharidosis III studies →UniQure Biopharma B.V. is the lead sponsor of 8 studies on the registry; 2 are open to participants now.
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Exclusion Criteria:
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).
Drug: rAAV2/5-hNAGLU
one-time brain intraparenchymal gene therapy dose
Also known as: AAV5
Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline
Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.
Time frame: Baseline until end of study (Month 66)
Number of Participants with presence of brain atrophy, white matter lesions and other lesions as assessed by cerebral MRI
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.
Time frame: Baseline until end of study (Month 66)
Ran from Sep 2013 to primary completion in Nov 2019, 74 months.
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This study is completed, as verified in Oct 2018. You cannot join it, but the record below documents what was studied.
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UniQure Biopharma B.V.