A Gaucher Disease Gene Therapy Trial With FLT201
ConditionGaucher Disease Type 1
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorSpur Therapeutics
About this trial
This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged ≥18 years at time of screening.
Clinical diagnosis of Gaucher disease type 1
Stable hemoglobin concentration at baseline
Stable platelet count at baseline
Disqualifiers
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
Trial design
Design model
Single group
Treatments tested in this trial
FLT201
GeneticFLT201 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.
Treatment groups
45 Participants
are divided into 1 treatment groupGroup A: FLT201Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
To evaluate the efficacy of FLT201
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
Time frame
1 YearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov