Eliglustat
Eliglustat appears in the pilot as an oral GCS inhibitor with a GD1 clinical-trial package. The source summary lists it as marketed for GD type 1, with PD relevance marked as uncertain. Source: 20240722_181748.
Trial Map
| trial / phase | source-captured population | design / treatment | key source note | source |
|---|---|---|---|---|
| P3 ENCORE | GD type 1 adults who had received ERT for 3 years or more and would switch to oral eliglustat | Randomized open-label non-inferiority, 12 months, n=160 | Eliglustat stability 85% versus imiglucerase 94%; oral eliglustat was non-inferior to imiglucerase. | 20240722_181748 |
| P3 ENGAGE | Untreated GD1 for past 9 months, no splenectomy symptoms | Eliglustat vs placebo, 9 months, n=40 | Primary outcome spleen volume; secondary hemoglobin, liver volume, platelet count. | 20240722_181748 |
| P3 EDGE | GD1 patients stable on twice-daily eliglustat, QD vs BID | n=171 | Source notes 8-year follow-up results published 2018 Lukina. | 20240722_181748, 20240722_181752 |
| P2 | Untreated GD1 adults with GBA1 enzyme deficiency and spleen/platelet/anemia criteria | Open-label single arm, 50 mg BID, 52 weeks, n=28 | Statistically significant improvements in hemoglobin, platelet count, spleen volume, liver volume, lumbar spine BMD, and biomarkers. | 20240722_181752 |
| P2 extension | Extension of the P2 study | One further year, n=20 | Improvements maintained and extended through 4 years. | 20240722_181752 |
| P1 SAD / MAD / food effect | Healthy-volunteer style early development rows | SAD 0.01 to 30 mg/kg; MAD 50, 200, or 350 mg BID | Only sparse row data visible in pilot. | 20240722_181752 |
Biomarker Context
The local table header immediately above eliglustat says “Glucosylceramide, GlcSph, a-syn”, but the visible eliglustat rows are mostly GD clinical trial outcomes rather than a PD biomarker package. Source: 20240722_181748.
Uncertainties Carried From Source
| issue | source |
|---|---|
| P3 EDGE row is split across photos; right-side design/outcome cells are clipped in 20240722_181748 and continued in 20240722_181752. | 20240722_181748, 20240722_181752 |
published 2018 Lukina is small but visually consistent in the source. | 20240722_181752 |