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Home › Conditions › Gaucher disease

Gaucher disease

0 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Gaucher disease

20 trials tracked for Gaucher disease: 14 in phase 3 or 4 and 1 with published results. The most-cited published study has 26 citations.

20Trials tracked
14Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 4 3 Phase 3 11 Phase 2 4 Other / NA 2
  1. Phase 3 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) Completed · 26 cited
  2. Phase 4 Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease Completed
  3. Phase 4 A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy Completed
  4. Phase 4 A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Completed
  5. Phase 3 Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies Completed
  6. Phase 3 A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease Completed
Show 14 more trials
  1. Phase 3 Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease Completed
  2. Phase 3 A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Completed
  3. Phase 3 Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase Completed
  4. Phase 3 A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease Completed
  5. Phase 3 Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase Completed
  6. Phase 3 Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease Completed
  7. Phase 3 A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease Completed
  8. Phase 3 Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial Completed
  9. Phase 2 Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) Completed
  10. Phase 2 A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy Completed
  11. Phase 2 A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients Completed
  12. Phase 2 A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease Completed
  13. N/A Auto-antibodies Prevalence and CD1 Role in Gaucher Disease Completed
  14. N/A Survey Study for Velaglucerase Alfa (VPRIV) in Japan Completed

Showing the 20 most-cited and recently-updated of 20 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Gaucher disease: what the trials found

Therapeutic management of Gaucher disease includes several enzyme replacement therapies and oral treatments. Velaglucerase alfa has been shown to reduce Bone Marrow Burden (BMB) scores by 3.0 at week 51 and 103 2 and further reductions in BMB score were noted in smaller cohorts 9. Treatment with velaglucerase alfa was associated with a reduction in bone pain interference scores of -1.286 at week 51 and -4.429 at week 103 2. Additionally, velaglucerase alfa (GA-GCB) demonstrated a 7.04% increase in platelet count from baseline to week 53 and reductions in normalized liver volume (-0.03%) and spleen volume (-5.56%) by week 51 14.

Taliglucerase alfa has been evaluated across multiple trials [3, 7, 8, 11, 12]. Plant cell expressed recombinant glucocerebrosidase (prGCD) demonstrated significant clinical improvements, including a reduction in spleen volume (-26.91 to -38.01; p<0.0001), reduction in liver volume (-10.48 to -11.11; p=0.0041), and an increase in hemoglobin (1.6 to 2.2; p=0.0010) 13.

Eliglustat tartrate was evaluated for stability, with over 80% of participants remaining stable for 52 weeks 4. In a long-term study involving eliglustat and GZ385660, bone pain levels were reported at baseline but significantly decreased by week 52, with no patients reporting bone crisis after the initial assessment period 6.

Other interventions include afegostat tartrate in multiple phases of clinical trials [15, 16, 17] and a digital engagement application (GD App) 1. Regarding safety for velaglucerase alfa, 95% of participants experienced treatment-emergent adverse events (TEAEs), with 20% experiencing at least one serious TEAE and 5% reporting infusion-related reactions 5.

Recent results — preliminary, needs further review

  • GA-GCB showed significant improvements in hemoglobin concentration (approx. 16-20% increase), platelet counts (approx. 78-139% increase), and liver volume reduction (approx. 28-42% decrease) in a Phase 2 study, though these findings are not yet corroborated 18.
  • Genetic analyses were conducted in a cohort of 60 patients, but results are not yet corroborated 20.

For the clinician treating this condition

  • Velaglucerase alfa is associated with reductions in bone marrow burden and bone pain interference scores [2, 9].
  • prGCD shows statistically significant improvements in spleen volume, liver volume, and hemoglobin levels 13.
  • Eliglustat tartrate demonstrates high stability over a 52-week period 4.

AI synthesis of 10 cited trials, updated Jun 25, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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