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

NCT02716246 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-04-24

No results posted yet for this study

Summary

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

Conditions

  • MPS IIIA
  • Sanfilippo Syndrome
  • Sanfilippo A
  • Mucopolysaccharidosis III

Interventions

DRUG

Adjuvant IM Therapy

The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy.

BIOLOGICAL

UX111

Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein.

DRUG

Prophylactic Immunomodulatory (IM) Therapy

DRUG

Optimized Prophylactic IM Therapy

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-04-25
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Spain

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02716246 on ClinicalTrials.gov