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Study identifier: NCT03315182 Synced from ClinicalTrials.gov · July 30, 2026
● Terminated

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Condition: Mucopolysaccharidosis Type 3 B  ·  Sponsor: Abeona Therapeutics, Inc

PhasePhase 1/Phase 2
Planned participants11
Who can joinAll sexes, N/A to no upper limit
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Contact details are listed per location below or on the registry record.

Always discuss trial participation with your own doctor first.

Locations (4)

Nationwide Children's HospitalColumbus, Ohio, United States
Armand-Trousseau HospitalParis, France
University Hospital Hamburg-EppendorfHamburg, Germany
Hospital Clinico Universitario de SantiagoSantiago de Compostela, Spain

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Source record: clinicaltrials.gov/study/NCT03315182