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A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 45

Last updated 2026-04-21

No results posted yet for this study

Summary

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.

Conditions

  • Gaucher Disease Type 1

Interventions

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.

Sponsors & Collaborators

  • Spur Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-07
Primary Completion
2028-07-01
Completion
2032-10-01
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07223944 on ClinicalTrials.gov