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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

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30 recruiting trials

for Lysosomal Diseases

Cystinosis and Mitochondrial Metabolism NCT07319091NA9 sites📍 Bron📍 Lille📍 Lyon Hospices Civils de Lyon · Tests: Mitochondrial metabolism Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI NCT04856059NA1 site📍 Toronto University Health Network, Toronto · Tests: Cardiac MRI, ECG/Holter and Blood Biomarkers Cardiac Structure and Function in MPS NCT07361536n/a3 sites📍 Orange📍 Minneapolis📍 St Louis Children's Hospital of Orange County Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study NCT06758648n/a1 site📍 München Technical University of Munich · Tests: Dynamic retinal vessel analysis (DVA), Biochemistry and immune phenotyping, Questionnaires (Patient reported outcomes),… Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH NCT02716246Phase 2, Phase 35 sites📍 Columbus📍 Pittsburgh📍 North Adelaide Ultragenyx Pharmaceutical Inc · Tests: UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease NCT06539624NA2 sites📍 Shanghai📍 Hangzhou The Children's Hospital of Zhejiang University School of Me… · Tests: EXG110 injection Parkinson's Disease in Type 1 Gaucher Disease NCT07886879n/a1 site📍 New York Icahn School of Medicine at Mount Sinai Retinal Hyperspectral Imaging in Neurodegenerative Diseases NCT07545473NA1 site📍 Melbourne Center for Eye Research Australia · Tests: Hyperspectral camera Early Trial of Allogeneic Hematopoietic Stem Cell Transplantation for Patients Who Will Receive a Kidney Transplant From the Same Donor NCT05508009Phase 1, Phase 21 site📍 Palo Alto Alice Bertaina · Tests: Cyclophosphamide 1200 mg/Kg, Fludarabine, Cyclophosphamide 100 mg/Kg, Total Body Irradiation, ATG Effects of Bisphosphonates on OI-Related Hearing Loss NCT04152551Phase 41 site📍 New York Hospital for Special Surgery, New York · Tests: Risedronate Oral Tablet A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) NCT07082725Phase 334 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: Nizubaglustat, Placebo Natural History of Pompe Disease NCT03564561n/a1 site📍 Garches Assistance Publique - Hôpitaux de Paris ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I NCT05682144Phase 12 sites📍 Oakland📍 Minneapolis Immusoft of CA, Inc. · Tests: Autologous Plasmablasts (B cells) Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC NCT04528355n/a1 site📍 Pittsburgh Paul Szabolcs · Tests: data collection Anderson-Fabry Disease Fitness Improvement Training NCT07495410NA1 site📍 Prague General University Hospital, Prague · Tests: Physiotherapy exercise programe Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease NCT05698901n/a1 site📍 Taipei Mackay Memorial Hospital · Tests: Agalsidase beta A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) NCT07282847Phase 1, Phase 29 sites📍 Phoenix📍 Irvine📍 Palo Alto AskBio Inc · Tests: AB-1009 (GAA Gene) A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease NCT06818838Phase 1, Phase 23 sites📍 Guangzhou📍 Taiyuan📍 Tianjin Lingyi Biotech Co., Ltd. · Tests: LY-M001 A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis NCT03952637Phase 1, Phase 21 site📍 Bethesda National Human Genome Research Institute (NHGRI) · Tests: AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) NCT04532047Phase 11 site📍 San Francisco University of California, San Francisco · Tests: Aldurazyme (laronidase) A Natural History Study in Children With a Type II Collagen Disorder With Short Stature NCT05408715n/a2 sites📍 Paris📍 Madrid Innoskel · Tests: Natural History Study Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques NCT07336394n/a1 site📍 Beijing Chinese Academy of Medical Sciences, Fuwai Hospital Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease NCT05843552n/a1 site📍 Minneapolis University of Minnesota · Tests: no intervention GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma NCT05992532n/a23 sites📍 Vitoria-Gasteiz📍 Almería📍 Palma de Mallorca Fundación Española de Hematología y Hemoterapía A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating NCT07591584NA1 site📍 Los Angeles Kiwi Health Inc · Tests: FODMAP-targeting digestive enzyme blend, Placebo A Study of Patients With Fabry Disease (US Specific) NCT06906367n/a8 sites📍 Birmingham📍 Little Rock📍 Atlanta Amicus Therapeutics · Tests: migalastat HCl, ERT Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients NCT05858606NA1 site📍 Montpellier University Hospital, Montpellier · Tests: Evaluation of the prevalence of truly (authentified) idiopathic short stature after multidisciplinary clinico-radiologi… A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C NCT05163288Phase 314 sites📍 Rochester📍 Parkville📍 Prague IntraBio Inc · Tests: N-Acetyl-L-Leucine, Placebo National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children NCT06985212n/a1 site📍 Paris Wladimir MAUHIN, Dr Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma NCT03721068Phase 12 sites📍 Atlanta📍 Chapel Hill UNC Lineberger Comprehensive Cancer Center · Tests: iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine
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