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

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NCT02716246 · readout ≤ 955 d

Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →. With Abeona Therapeutics, Inc.

Phase

Phase 2/3

Status

Recruiting

Study type

Interventional

Design

Non randomized · None · Treatment

Enrollment

36estimated

Sites

Countries

Australia, Spain, United States

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

2016-04-25

actual

When the study began enrolling

Primary completion

Mar 2029

estimated

The date the last participant is measured for the primary outcome — the readout window

Study completion

Mar 2029

estimated

The whole study's end, after follow-up

First posted

2016-03-23

estimated

When this record first appeared on the registry

Results posted

—

When the sponsor posted results to the registry

Record updated

2026-07-17

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Conditions

MPS IIIA
Sanfilippo Syndrome
Sanfilippo A
Mucopolysaccharidosis III

Interventions

Biological: UX111 — also filed as scAAV9.U1a.hSGSH, ABO-102, rebisufligene etisparvovec
Drug: Prophylactic Immunomodulatory (IM) Therapy
Drug: Optimized Prophylactic IM Therapy
Drug: Adjuvant IM Therapy

Primary outcome

what the primary-completion date above is the date OF

Cerebrospinal Fluid (CSF) Heparan Sulfate (HS) (Disaccharide) Exposure

measured Up to Month 24 Visit

Publications

PMID 27331076 — cited as background
PMID 29064732 — cited as background
PMID 32884151 — linked by the registry

Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.

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