NCT02716246Phase 2, phase 3Recruiting

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

Study sponsor: Ultragenyx Pharmaceutical Inc · RARE

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2029-03Estimated

Enrollment · participants36Estimated

Source updated2026-09-22Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-06-11 · 1 field changed

Primary completion date changed

− Before

~~Jul 2027~~

+ After

Mar 2029

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Study details

Conditions studied

MPS IIIA; Sanfilippo Syndrome; Sanfilippo A; Mucopolysaccharidosis III

Treatments or interventions studied

UX111BiologicalAlso known as scAAV9.U1a.hSGSH, ABO-102, rebisufligene etisparvovec
Prophylactic Immunomodulatory (IM) TherapyDrug
Optimized Prophylactic IM TherapyDrug
Adjuvant IM TherapyDrug

Primary outcomes · main study measures

measure: Cerebrospinal Fluid (CSF) Heparan Sulfate (HS) (Disaccharide) Exposure; description: Exposure is defined as the time-normalized area under the curve (AUC) of the percentage reduction from baseline.; timeFrame: Up to Month 24 Visit

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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