Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE3
45 participants
INTERVENTIONAL
2026-07-01
2032-10-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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FLT201
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
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Spur Therapeutics
INDUSTRY
Responsible Party
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Central Contacts
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Other Identifiers
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2025-520765-50-00
Identifier Type: CTIS
Identifier Source: secondary_id
FLT201-03
Identifier Type: -
Identifier Source: org_study_id
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