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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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.