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Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

NCT03300453 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2019-12-02

No results posted yet for this study

Summary

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.

Conditions

  • Sanfilippo Syndrome B

Interventions

DRUG

rAAV2/5-hNAGLU

one-time brain intraparenchymal gene therapy dose

Sponsors & Collaborators

  • Venn Life Sciences

    collaborator OTHER

  • Institut Pasteur

    collaborator INDUSTRY

  • UniQure Biopharma B.V.

    lead INDUSTRY

Principal Investigators

  • Kumaran Deiva, MD · Hopitaux Universitaires Paris-Sud

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
60 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-09-17
Primary Completion
2019-11-27
Completion
2019-11-27

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03300453 on ClinicalTrials.gov