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

NCT03612869 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2021-08-31

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis Type IIIA

Interventions

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.

Sponsors & Collaborators

  • LYSOGENE

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-17
Primary Completion
2022-03-31
Completion
2022-03-31
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

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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 NCT03612869 on ClinicalTrials.gov