These trials completed more than 12 months ago and should have reported results.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Exempt, with results 2015-001985-25 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 (MP... 2020-06-19 not-yet-due
Ongoing 2017-003083-13 A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syn... not-yet-due
Other 2022-002762-33 A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB... not-yet-due