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Study identifier: NCT02716246 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Condition: MPS IIIA · Sanfilippo Syndrome · Sanfilippo A  ·  Sponsor: Ultragenyx Pharmaceutical Inc

PhasePhase 2/Phase 3
Planned participants36
Who can joinAll sexes, N/A to no upper limit
Healthy volunteersNo

About this study

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

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

Talk to the study team

Patients Contact: Trial Recruitment  ·  1-888-756-8657  ·  trialrecruitment@ultragenyx.com

HCPs Contact: Medical Information  ·  1-888-756-8657  ·  medinfo@ultragenyx.com

Always discuss trial participation with your own doctor first.

Locations (5)

Nationwide Children's HospitalColumbus, Ohio, United StatesCompleted
Children's Hospital of PittsburghPittsburgh, Pennsylvania, United StatesCompleted
Women's and Children's HospitalNorth Adelaide, South Australia, AustraliaCompleted
Vall d'Hebron Barcelona Hospital CampusBarcelona, Barcelona, SpainRecruiting
Hospital Clínico Universitario de SantiagoSantiago de Compostela, SpainRecruiting

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Source record: clinicaltrials.gov/study/NCT02716246