NCT03612869

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

• Status: Unknown
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: LYSOGENE · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

Summary

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

Detailed description

The study is interventional, single arm and multi-center. Evolution under treatment will be compared to expected natural evolution based on natural history studies.

Conditions

• Mucopolysaccharidosis Type IIIA

Interventions

Type	Name	Description
DRUG	LYS-SAF302	Treatment will involve direct injections of the investigational product into both sides of the brain through image-guided tracks, in a single neurosurgical session.

Timeline

• Start date: 2018-12-17
• Primary completion: 2022-03-01
• Completion: 2022-03-01
• First posted: 2018-08-02
• Last updated: 2021-08-31

Locations

8 sites across 5 countries: United States, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study