Status:
COMPLETED
Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
Lead Sponsor:
UniQure Biopharma B.V.
Collaborating Sponsors:
Venn Life Sciences
Institut Pasteur
Conditions:
Sanfilippo Syndrome B
Eligibility:
All Genders
18-60 years
Phase:
PHASE1
PHASE2
Brief 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.
Detailed Description
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. F...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Age: 18 months up to 60 months (5th birthday);
- Onset of clinical manifestations related to mucopolysaccharidosis type IIIB (MPSIIIB);
- NAGLU activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls;
- Patient affiliated to, or covered by a French social security regimen, or European patients with European Health Insurance Card;
- Family understanding the procedure and the informed consent;
- Signed informed consent by both parents or legal representative;
- Vital laboratory parameters within normal range.
- Exclusion Criteria:
- Presence of brain atrophy on baseline MRI judged on a cortico-dural distance of more than 0.6 cm;
- Any condition that would contraindicate general anesthesia;
- Any other permanent medical condition not related to MPSIIIB that could contraindicate the study participation;
- No independent walking (ability to walk without help);
- Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection (sleep and mood regulators are accepted);
- Any condition that would contraindicate treatment with Modigraf®, Cellcept® and prednisolone (Solupred® and Solumedrol®).
Exclusion
Key Trial Info
Start Date :
September 17 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 27 2019
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT03300453
Start Date
September 17 2013
End Date
November 27 2019
Last Update
December 2 2019
Active Locations (1)
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1
Hopitaux Universitaires Paris-Sud
Paris, Le Kremlin-Bicetre Cedex, France, 94275