Last updated 2 days ago
A Gaucher Disease Gene Therapy Trial With FLT201
45 patients around the world
Available in Argentina, United States, Brazil
Spur Therapeutics
4Research sites
45Patients around the world
This study is for people with
Gaucher disease
Gaucher disease type 1
Requirements for the patient
From 18 Years
All Gender
Medical requirements
- Aged ≥18 years at time of screening.Clinical diagnosis of Gaucher disease type 1.Stable hemoglobin concentration at baseline.Stable platelet count at baseline.Receiving ERT or SRT without interruption for at least 2 years.
- Diagnosed or suspected Gaucher disease type 2 or type 3.Positive for AAVS3 neutralizing antibodies.Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study.Positive pregnancy test or lactating.History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.History of receiving any gene therapy or cell therapy.History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply.
Sites
Hospital Italiano de Buenos Aires - CABA, Buenos Aires
Clínica Universitaria Privada Reina Fabiola - Córdoba
Hospital de Clínicas de Porto Alegre (HCPA)
Hospital Militar Central
StudyGALILEO-3
SponsorSpur Therapeutics
Study typeInterventional
Conditions
Gaucher disease type 1
Requirements
From 18 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT07223944
