Browse recruiting and upcoming clinical trials for Gaucher disease.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
About this listHide
ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Who can join AIAdults 18–60 with genetically confirmed Gaucher disease type I (biallelic GBA1 mutations with low GCase activity).
What is being studied AITests safety, preliminary efficacy, biodistribution, immunogenicity, pharmacodynamics, and viral shedding after one LY-M001 infusion.
Who can join AIPeople aged 2 months–55 years with non‑malignant disorders treatable by hematopoietic stem‑cell transplant (examples: immune, metabolic, hereditary anemias, inflammatory conditions).
What is being studied AITests reduced-intensity conditioning before stem cell transplant vs standard myeloablative conditioning.
Who can join AIPeople with Gaucher disease (GD1, GD2, or GD3), including neuronopathic forms, refractory skeletal disease, or Gaucher–Parkinson overlap.
What is being studied AICollects clinical data, imaging, lung tests, bone scans, and blood biomarkers over 36 months.
Who can join AIAdults (18–99) with unexplained splenomegaly, prior splenectomy of unknown cause, or thrombocytopenia being screened for Gaucher disease and ASMD.
What is being studied AIPrevalence of Gaucher disease and ASMD using dried blood spot (DBS) testing in targeted patients.
Who can join AIPeople aged 2 months–60 years with non-malignant disorders (e.g., primary immunodeficiency, bone marrow failure, inherited metabolic disorders, hereditary anemias, inflammatory conditions) undergoing UCB, bone marrow, or peripheral blood stem cell transplant with a reduced-intensity conditioning regimen.
What is being studied AICollects clinical outcomes after alemtuzumab-based reduced-intensity conditioning stem cell transplants.
Who can join AIPeople with rare genetic neurodegenerative disorders (for example MLD, Krabbe disease, ALD, Batten disease, Gaucher disease, Niemann‑Pick disease, and others).
What is being studied AIRegular clinical evaluations, developmental testing, MRI, and collection of blood, CSF, and urine biomarkers.
Who can join AIAdults (18–75) with Gaucher disease type 1 who are treatment‑naive or stable on ERT/SRT; the study measures oxidative stress and inflammation.
What is being studied AIMeasures blood oxidative stress and inflammatory biomarkers including C5a and hepcidin.
Who can join AIPeople with lysosomal storage disorders (including Gaucher), carriers, family members, healthy people, and people with Parkinson disease.
What is being studied AICollects genetic, biochemical, and clinical data to study disease features.
Who can join AIPeople (prenatally or postnatally diagnosed) with Mucopolysaccharidosis I, II, IV A, VI, VII; infantile-onset Pompe; neuronopathic Gaucher; or Wolman disease, ages 0–64.
What is being studied AICollects clinical and laboratory data to describe natural history and prenatal outcomes of lysosomal diseases.
ClinicalTrials.gov returned these when we searched for this condition, but we could not confirm from the registry records that they are intended for people with it. Check each trial’s eligibility criteria, and ask the study team if you are unsure.
Who can join AIWomen 18–75 with recurrent or refractory epithelial ovarian, fallopian tube, or primary peritoneal carcinoma after at least two prior systemic regimens.
What is being studied AITests safety, tolerability, and preliminary anti-tumor activity of EB-DUALNK dual-target CAR-NK cells.
Who can join AIAdults 18–75 with advanced unresectable, metastatic, or recurrent solid tumors for which standard curative therapy is unavailable, not tolerated, or has failed.
What is being studied AITests biomarker-guided use of autologous dual-target CAR-T cells for treating advanced solid tumors.
Who can join AIChildren under 18 with GD2-positive bone or soft-tissue sarcomas (eg, osteosarcoma, Ewing, rhabdomyosarcoma) that progressed after 1st-line chemotherapy.
What is being studied AIDinutuximab beta combined with investigator-chosen chemotherapy.
Who can join AIAdults (≥18) with extensive-stage lung cancer or stage IV non–small cell lung cancer who are platinum‑refractory and have received PD‑1/PD‑L1 therapy.
What is being studied AITests the safety and dosing of autologous GD2-targeted CAR T-cell therapy in lung cancer.
Who can join AIPeople aged 6 months to 30 years with relapsed or refractory central nervous system (CNS) tumors such as medulloblastoma, embryonal tumors, high-grade glioma, DMG/DIPG, and similar brain tumors.
What is being studied AITests safety, dosing and behavior of iC9-GD2 CAR T‑cell therapy in relapsed/refractory brain tumors
Who can join AIPeople (age ≥12 months) with newly diagnosed, high‑risk (metastatic) GD2‑positive Ewing sarcoma or Ewing‑like translocation‑positive small round blue cell sarcoma.
What is being studied AITests dinutuximab beta added to standard VDC/IE chemotherapy in GD2-positive Ewing sarcoma.