CLINICAL TRIAL PROFILE · UPDATED 2026-09-29

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

NCT02716246 · Phase 2 · Recruiting

PHASEPhase 2

STATUSRecruiting

ENROLLMENT36

EST. COMPLETION2029-03

Conditions

MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis III

Interventions

UX111Prophylactic Immunomodulatory (IM) TherapyOptimized Prophylactic IM TherapyAdjuvant IM Therapy

Study details

SPONSORUltragenyx Pharmaceutical Inc

START DATE2016-04-25

Related company profiles

Ultragenyx

RARE

Primary source

ClinicalTrials.gov record NCT02716246 ↗

Trial status and dates can change. Confirm enrollment and eligibility against the linked ClinicalTrials.gov record.

Evidence & limits

Source: Open the source record

Record identifier: NCT02716246
Event / action date: Not separately retained
Source update date: Not separately retained
Platform retrieval / processing date: Not separately retained

What it supports. Study information reported by the sponsor or investigator, including status, phase, enrollment, and recorded dates.

Limits. Registration or completion does not establish clinical success or approval. Sponsor-name associations can be ambiguous. Dates may be estimated; verify the live study record.

Connection basis. Company sponsor aliases and normalized lead-sponsor containment; the NCT identifier identifies the study.

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