A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-510392-66-00

Acronym

LTFU-ABO-102

Enrollment

Registered

2024-02-07

Start date

2020-11-23

Completion date

Unknown

Last updated

2025-10-20

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: Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive raw score

Detailed description

Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure, CSF HS percentage change from baseline in prior trial, BSITD-III Receptive Communication raw score, BSITD-III Expressive Communication raw score, Total cortical volume (cm 3) annualized percentage change from baseline in prior trial, Survival

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: Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive raw score	—

Secondary

Measure	Time frame
Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure, CSF HS percentage change from baseline in prior trial, BSITD-III Receptive Communication raw score, BSITD-III Expressive Communication raw score, Total cortical volume (cm 3) annualized percentage change from baseline in prior trial, Survival	—

Countries

Spain

Outcome results

None listed

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