MedTrace Logo

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799 · Status: ENROLLING_BY_INVITATION · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2023-08-09

No results posted yet for this study

Summary

This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment with AX 250 administered by ICV infusion every other week. Subjects will be evaluated for neurocognitive function, communication, adaptive behavior, quality of life, imaging characteristics and biochemical markers of disease burden. Safety will be assessed by adverse events, clinical labs, and physical exams.

Conditions

  • MPS III B

Interventions

DRUG

AX 250

biweekly infusion by intracerebroventricular (ICV) infusion

Sponsors & Collaborators

  • Allievex Corporation

    lead INDUSTRY

Principal Investigators

  • Medical Director · Allievex Corporation

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-02
Primary Completion
2027-10-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States
  • Colombia
  • Germany
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05492799 on ClinicalTrials.gov