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A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1

Status: Recruiting

The purpose of this study is to evaluate the safety and effectiveness of FLT201, an investigational gene therapy for adults with Gaucher disease type 1. Participants will receive a one-time infusion of FLT201, continue their current treatment for a short time, and complete regular study visits and health assessments over a 5-year follow-up period. Researchers will study the safety of FLT201, how well it works, and how long its effects last.

I'm interested

Sex: Male or Female
Age Group: 18 years and over
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 18 years of age or older
• diagnosed with Gaucher disease type 1
• currently receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years
• see the ClinicalTrials.gov listing for complete inclusion criteria
Exclusion Criteria:

• diagnosis or suspected diagnosis of Gaucher disease type 2 or type 3
• previous gene therapy, cell therapy, or organ/bone marrow transplant
• pregnant or breastfeeding
• see the ClinicalTrials.gov listing for complete exclusion criteria
Interventions:

Genetic: FLT201

Conditions:

Rare Diseases, Blood Disorders, Digestive & Liver Health

Keywords:

Clinics and Surgery Center (CSC), Enzyme replacement therapy, ERT, Gaucher disease, Gene therapy, Rare disease

Study Contact: Brenda Diethelm-Okita - dieth001@umn.edu
Principal Investigator: Chester Whitley, MD, PhD
Phase: PHASE3
IRB Number: STUDY00025671
See this study on ClinicalTrials.gov

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