Skip to main content

Table 2 Baseline clinical values prior to eliglustat treatment in each trial

From: Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1

Trial N Patient Population Spleen Volumea (MN) Liver Volume (MN) Hemoglobin (g/dL) Platelet Count (×109/L)
    Mean (min, max) Mean (min, max) Mean (min, max) Mean (min, max)
Phase 2 Open-label (NCT00358150) 26 Treatment-naïve 20.0 (8.2, 59.7) 1.8 (0.8, 3.9) 11.1 (8.1, 14.6) 66 (39, 106)
ENGAGE Phase 3 Randomized, Placebo-controlled (NCT00891202) 40 Treatment-naïve 13.4 (5.9, 28.4) 1.4 (0.9, 2.2) 12.1 (7.9, 15.3) 73 (36, 126)
ENCORE Phase 3 Randomized, Imiglucerase-controlled (NCT00943111) 157 Stable after ≥3 years of ERT 3.0 (1.1, 5.3) 0.9 (0.5, 1.7) 13.7 (10.7, 17.7) 201 (104, 368)
EDGE Phase 3 Randomized Dosing Regimen (NCT001074944) 170 Mostly ERT switch patients 4.5 (0.8, 11.3) 1.0 (0.6, 1.9) 13.4 (9.0, 17.1) 179 (72, 721)
  1. ERT enzyme replacement therapy, MN multiples of normal
  2. For ENGAGE and ENCORE: baseline represents values at trial entry for patients treated with eliglustat during the primary analysis and values at extension entry for patients first treated with placebo or imiglucerase
  3. aExcludes splenectomized patients in ENCORE and EDGE clinical trials