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Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

NCT03315182 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2022-05-05

No results posted yet for this study

Summary

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

Conditions

  • Mucopolysaccharidosis Type 3 B

Interventions

BIOLOGICAL

rAAV9.CMV.hNAGLU

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein.

Sponsors & Collaborators

  • Abeona Therapeutics, Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-10-16
Primary Completion
2022-04-07
Completion
2022-04-07
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Spain

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 NCT03315182 on ClinicalTrials.gov