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Gene Therapy Trial Report

Summary

A Gaucher Disease Gene Therapy Trial With FLT201


NCTID NCT07223944 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Gaucher Disease Type 1
Disease Ontology Term DOID:0110957
Compound Name FLT201
Compound Alias avigbagene parvec
Sponsor Spur Therapeutics
Funder Type Industry
Recruitment Status 🔄
Enrollment Count 45 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GBA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVS3
Editor Type none
Dose 1 4.5E11 vg/kg

Study Record Dates


Current Stage Phase3
Submit Date 2025-10-30
Completion Date 2032-10-01
Last Update 🔄 2026-04-21

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key 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 Key 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 3
Locations 🔄 United States

Regulatory Information


Has US IND True
FDA Designations Regenerative Medicine Advanced Therapy
Recent Updates Trial initiation expected 2H26

Resources/Links