A Gaucher Disease Gene Therapy Trial With FLT201

Purpose

This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.

Condition

  • Gaucher Disease Type 1

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
FLT201 		FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
  • Genetic: FLT201
    FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.

Recruiting Locations

Duke University Medical Center
Durham, North Carolina 27710

University of Texas Southwestern
Dallas, Texas 75390

Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia 22030-6066
Contact:
Lauren Noll
(703) 2616220
lnoll@ldrtc.org

More Details

Status
Recruiting
Sponsor
Spur Therapeutics

Study Contact

Spur Clinical Trials Contact
+44 (0)1438 906870
clinicaltrials@spurtherapeutics.com