Gaucher disease
MONDO:0018150Gaucher disease (GD) is a lysosomal storage disorder encompassing three main forms (types 1, 2 and 3), a fetal form and a variant with cardiac involvement (Gaucher disease - ophthalmoplegia - cardiovascular calcification or Gaucher-like disease).
Also known as: Gaucher disease, Gaucher syndrome, acid beta-glucosidase deficiency, glucocerebrosidase deficiency, glucocerebrosidosis, glucosylceramidase deficiency, glucosylceramide beta-glucosidase deficiency, lipoid histiocytosis (kerasin type)
46 clinical trials for this condition and its sub-types, 46 tagged with Gaucher disease itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Gaucher disease
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Gaucher disease type I 12 trials
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Gaucher disease type III 12 trials
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Gaucher disease type II 5 trials
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Gaucher disease perinatal lethal 0 trials
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Triple-Attack CAR-T cells take aim at stubborn neuroblastoma
Disease control Not yet recruitingThis clinical trial tests a new type of immunotherapy called 4SCAR-T, which uses a patient's own immune cells engineered to recognize and attack three different markers (GD2, PSMA, and CD276) on neuroblastoma cancer cells. The study enrolls children and adults (ages 1 to 65) with…
Phase 1/2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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New pill aims to tackle rare genetic disorder affecting brain and body
Disease control Not yet recruitingThis study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…
Phase 2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
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Gene therapy injection aims to control rare brain disease in children
Disease control Not yet recruitingThis early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure …
Phase 1/2 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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Do patients understand their home infusion guides? new survey aims to find out
Knowledge-focused Not yet recruitingThis survey will ask about 60 people—patients with Gaucher disease, their caregivers, and home infusion nurses—how well they understand and use educational materials for at-home VPRIV infusions. The materials include an infusion diary, a guide, and an emergency plan. The goal is …
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:03 UTC