NCT06545136

Brief Summary

This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
75

participants targeted

Target at P75+ for phase_1

Timeline
36mo left

Started May 2024

Longer than P75 for phase_1

Geographic Reach
4 countries

4 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress40%
May 2024May 2029

Study Start

First participant enrolled

May 13, 2024

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

July 29, 2024

Completed
11 days until next milestone

First Posted

Study publicly available on registry

August 9, 2024

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2029

Last Updated

November 4, 2025

Status Verified

October 1, 2025

Enrollment Period

5 years

First QC Date

July 29, 2024

Last Update Submit

October 31, 2025

Conditions

Gaucher Disease, Type 1

Outcome Measures

Primary Outcomes (1)

  • Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)

    Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded per CTCAE version 5.0 (or later)

    Week 38 to Month 60

Study Arms (1)

Dosed with FLT201

EXPERIMENTAL
Genetic: FLT201

Interventions

FLT201GENETIC

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.

Dosed with FLT201

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • \. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy \[SRT\]).
  • \. Participants able to give full informed consent and able to comply with all requirements of the trial.

You may not qualify if:

  • n/a

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-6066, United States

Location

Hospital de Clinicas de Porto Alegre (HCPA)

Porto Alegre, Brazil

Location

Hospital Quironsalud Zaragoza

Zaragoza, Spain

Location

Salford Royal Hospital

Salford, United Kingdom

Location

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 29, 2024

First Posted

August 9, 2024

Study Start

May 13, 2024

Primary Completion (Estimated)

May 1, 2029

Study Completion (Estimated)

May 1, 2029

Last Updated

November 4, 2025

Record last verified: 2025-10

Locations

GB(1)US(1)BR(1)ES(1)