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 Syndrome Type B)

NCT03784287 Phase 2 UNKNOWN

The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

Details

Lead sponsor	Allievex Corporation
Phase	Phase 2
Status	UNKNOWN
Enrolment	20
Start date	2018-02-19
Completion	2025-02

Conditions

• Mucopolysaccharidosis Type IIIB
• MPS III B

Interventions

• AX 250

Primary outcomes

• Evaluate the Long-term safety and tolerability of AX 250 administered in up to 33 subjects with MPS IIIB by assessing the number of participants with abnormal clinical laboratory values and/or Adverse Events related to the treatment — Entire study period, up to 240 weeks
• Evaluating the impact of long-term AX 250 treatment on cognitive function in up to 33 patients with MPS IIIB as assessed by developmental quotient (DQ) — Entire study period, up to 240 weeks

Countries

United States, Colombia, Germany, Spain, Taiwan, Turkey (Türkiye), United Kingdom