MedTrace Logo

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2023-07-25

Study results available
· View outcomes & findings →

Summary

Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein

Conditions

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

Interventions

DRUG

ABO-102

Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10\^13 vg/kg

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-18
Primary Completion
2022-03-10
Completion
2022-03-10
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Spain

Study Locations

More Related Trials

Entities

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 NCT04088734 on ClinicalTrials.gov