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A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2020-08-06

No results posted yet for this study

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

  • MPS III B
  • Mucopolysaccharidosis Type IIIB

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

  • Allievex Medical Monitor · Allievex Corporation

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-04-30
Primary Completion
2020-06-24
Completion
2020-07-31

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 NCT02754076 on ClinicalTrials.gov