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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
Not yet recruiting
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 2025-11-03

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
Locations

Regulatory Information


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

Resources/Links