Trials / Completed
CompletedNCT02754076
A Treatment Study of Mucopolysaccharidosis Type IIIB
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)
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 23 (actual)
- Sponsor
- Allievex Corporation · Industry
- Sex
- All
- Age
- 1 Year – 10 Years
- Healthy volunteers
- Not accepted
Summary
The study's primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | AX 250 | Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2) |
Timeline
- Start date
- 2016-04-01
- Primary completion
- 2020-06-24
- Completion
- 2020-07-31
- First posted
- 2016-04-28
- Last updated
- 2020-08-06
Locations
7 sites across 7 countries: United States, Colombia, Germany, Spain, Taiwan, Turkey (Türkiye), United Kingdom
Source: ClinicalTrials.gov record NCT02754076. Inclusion in this directory is not an endorsement.