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Clinical trial

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT02754076 is registered in ClinicalTrials.gov with Allievex Corporation as lead sponsor.

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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Lead sponsor
Allievex Corporation
Phase
Phase 1/2
Overall status
Completed
Start date
April 30, 2016
Completion date
July 31, 2020
Enrollment
23 (actual)
Sites
7

Conditions

As the registry lists them; a condition with its own page is linked.

mps iii b, mucopolysaccharidosis type iiib