This multicenter Phase 1/2 study by Spur Therapeutics provides long-term follow-up for participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants are followed for 5 years after treatment; eligibility is open to those who previously received FLT201, including those who may have needed to restart or begin enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), and who can give full informed consent and comply with trial requirements. The trial is active but no longer recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * 1\. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy \[SRT\]). * 2\. Participants able to give full informed consent and able to comply with all requirements of the trial. Exclusion Criteria: * n/a