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

PHASE2/PHASE3UnknownINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

December 17, 2018

Primary Completion Date

March 31, 2022

Study Completion Date

March 31, 2022

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.

Trial Locations (8)

1000

Amsterdam UMC, Amsterdam

20246

University Medical Center Hamburg-Eppendorf, Hamburg

55455

University of Minnesota, Minneapolis

75012

Armand Trousseau Public Hospital, Paris

77030

Baylor college of medicine / Texas children's hospital, Houston

92868

CHOC Children's, Orange

10065-4897

Weill Cornell Medical College, New York

Unknown

Great Ormond Street Hospital, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

LYSOGENE

INDUSTRY