NCT04088734

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

A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is an open-label, single dose clinical trial. All participants will receive 3 X 10\^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Conditions

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

Interventions

Type	Name	Description
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

Timeline

• Start date: 2019-09-18
• Primary completion: 2022-03-10
• Completion: 2022-03-10
• First posted: 2019-09-13
• Last updated: 2023-07-25
• Results posted: 2023-07-25

Locations

3 sites across 3 countries: United States, Australia, Spain

Regulatory

• FDA-regulated drug study