A Phase 3 interventional study of FLT201 in Gaucher Disease Type 1, sponsored by Spur Therapeutics. Recruiting at 32 sites in 10 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Spur Therapeutics · Phase 3, Interventional, and Treatment
Among 92 studies in Gaucher Disease with a site in United States — 20 open to participants now, 27 under way, 2 of them phase 3. Gaucher Disease · United States.
Among the 92 studies in Gaucher Disease with a site in United States, this one is the 12th largest by planned enrollment of the 51 interventional ones, is one of 3 that started in 2026, is one of 10 in phase 3, reaches primary completion later than 13 of the 25 other live ones, and was the 90th registered.
177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.
This study's planned enrollment of 45 is above the median of 20 across 100 interventional studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →20 of the 92 studies in Gaucher Disease with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 26 Gaucher Disease studies recruiting now, 11 (42%) set no upper age limit and 11 (42%) stop at 75 or younger; 15 take children under 18; 4 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →92 studies; the count open now beside each. This study's row is marked.
First in 1988; this study’s year is marked. 3 started in 2026.
Median enrollment 30 across the 51 interventional studies, 60 across the 39 observational. Of the 50 that completed or were terminated, 20 (40%) have results posted.
45 of 92 have an industry lead sponsor. The 10 most frequent:
Spur Therapeutics is the lead sponsor of 8 studies on the registry; 1 is open to participants now.
Spur Therapeutics has 3 studies filed under Gaucher Disease — fewer than ten, so there is no comparison to draw.
5 of the studies in Gaucher Disease with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
| NCT02437396University of MinnesotaOxidative Stress and Inflammatory Biomarkers in Gaucher Disease | 2026-12-31 |
| NCT05586243University of MinnesotaMAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3) | 2026-12-31 |
| NCT05843552University of MinnesotaExtracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease | 2026-12-31 |
| NCT04411654Prevail TherapeuticsPhase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | 2027-02 |
Among the 92 studies in Gaucher Disease with a site in United States, from their registry updates:
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.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
Genetic: 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.
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 Year
Started Apr 2026 (actual). Primary completion is estimated for Jul 2028, 26 months after the start.
Of 11 phase 3 interventional studies under Gaucher Disease, half reached primary completion within 25 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 57. This study's planned 26 months is close to that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Spur Therapeutics