Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-510032-37-00

Acronym

ABT-001

Enrollment

Registered

2024-02-07

Start date

2017-04-18

Completion date

Unknown

Last updated

2025-12-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis type IIIA

Brief summary

Safety: Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs), Efficacy: Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure

Detailed description

Bayley Scales of Infant and Toddler Development–Third edition (BSITD- III) Cognitive raw score, CSF ganglioside type 2 (GM2) exposure, CSF ganglioside type 3 (GM3) exposure, CSF HS percentage change from baseline, BSITD-III Receptive Communication raw score, BSITD-III Expressive Communication raw score, Total cortical volume (cm 3) percentage change from baseline

Interventions

DRUGABO-102

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Safety: Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs), Efficacy: Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure	—

Secondary

Measure	Time frame
Bayley Scales of Infant and Toddler Development–Third edition (BSITD- III) Cognitive raw score, CSF ganglioside type 2 (GM2) exposure, CSF ganglioside type 3 (GM3) exposure, CSF HS percentage change from baseline, BSITD-III Receptive Communication raw score, BSITD-III Expressive Communication raw score, Total cortical volume (cm 3) percentage change from baseline	—

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026