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A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

NCT03784287 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2023-01-18

No results posted yet for this study

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).

Conditions

  • Mucopolysaccharidosis Type IIIB
  • MPS III B

Interventions

DRUG

AX 250

Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)

Sponsors & Collaborators

  • Allievex Corporation

    lead INDUSTRY

Principal Investigators

  • Medical Monitor, MD · Allievex Corporation

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-02-19
Primary Completion
2025-01-31
Completion
2025-02-28
FDA Drug
Yes

Countries

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

Study Locations

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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 NCT03784287 on ClinicalTrials.gov