drugset / Trial / NCT03784287
A Treatment Extension Study of Mucopolysaccharidosis Type IIIB
NaSingle-groupOpen-labelTreatment
Summary
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).
Timeline
- Start
- 2018-02-19
- Primary completion
- 2025-01
- Completion
- 2025-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AX 250 | Protein / enzyme biologic | 300 mg | Other |