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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 Metabolism, Inborn Errors

Echocardiographic Characterization of Cardiac Amyloidosis NCT05760287n/a1 site📍 Roma Fondazione Policlinico Universitario Agostino Gemelli IRCCS An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease NCT06272149EARLY Phase 11 site📍 Shanghai Xinhua Hospital, Shanghai Jiao Tong University School of Me… · Tests: VGN-R08b Caregiving Networks Across Disease Context and the Life Course NCT05007990n/a1 site📍 Bethesda National Human Genome Research Institute (NHGRI) A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease NCT06328608Phase 2, Phase 312 sites📍 Phoenix📍 Atlanta📍 Iowa City Chiesi Farmaceutici S.p.A. · Tests: PRX-102 1 mg/kg every two weeks Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168n/a1 site📍 Sioux Falls Sanford Health Natural History Study of Patients With Hypophosphatasia (HPP) NCT02237625n/a1 site📍 Durham Duke University Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia NCT07473843Phase 33 sites📍 St Louis📍 Viborg📍 Warsaw Arrowhead Pharmaceuticals · Tests: Zodasiran A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD) NCT06192576n/a6 sites📍 Chicago📍 Cincinnati📍 Columbus Sanofi · Tests: Olipudase alfa LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study NCT06217588n/a1 site📍 Philadelphia University of Pennsylvania · Tests: Demographics, diagnosis type, genotype, lipid profile, renal function profile, treatment allocation, ophthalmology exam… Rare Kidney Stone Consortium Patient Registry NCT00588562n/a4 sites📍 Rochester📍 Rochester📍 New York Mayo Clinic An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis NCT05335603n/a1 site📍 Hvidovre University of Copenhagen Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells NCT06967831n/a2 sites📍 Düsseldorf📍 Berlin Charite University, Berlin, Germany · Tests: skin biopsy, generation of iPSCs, blood drawing, off-label compassionate drug use Natural History Study of Parathyroid Disorders NCT04969926n/a1 site📍 Bethesda National Institute of Diabetes and Digestive and Kidney Dis… A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101) NCT07240896EARLY Phase 11 site📍 Hefei DSciLab Co., Ltd. · Tests: Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose, Group 3: DSL101 High dose An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia NCT06712823Phase 213 sites📍 Minneapolis📍 Morehead City📍 Philadelphia Crinetics Pharmaceuticals Inc. · Tests: atumelnant (CRN04894) A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria NCT07241234Phase 15 sites📍 Indianapolis📍 Pittsburgh📍 Dallas Agios Pharmaceuticals, Inc. · Tests: AG-181 Danon Disease Natural History Study NCT06214507n/a16 sites📍 Phoenix📍 La Jolla📍 Aurora Rocket Pharmaceuticals Inc. · Tests: No intervention Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies NCT06844877n/a1 site📍 Pisa IRCCS Fondazione Stella Maris KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases NCT06451757Phase 310 sites📍 Boston📍 Cleveland📍 Houston Khondrion BV · Tests: Sonlicromanol, Placebo A Long-Term Study of JNT-517 in Participants With Phenylketonuria NCT06628128Phase 312 sites📍 Gainesville📍 Tampa📍 Portland Otsuka Pharmaceutical Development & Commercialization, Inc. · Tests: JNT-517 Natural History of Wilson Disease NCT03334292n/a6 sites📍 New Haven📍 Orlando📍 Houston Yale University A Phase 2 Trial of Dotinurad in Xanthine Oxidase Inhibitor (XOI) Intolerant/Uricase Failure Gout Participants NCT07535034Phase 241 sites📍 Birmingham📍 Phoenix📍 Tucson Crystalys Therapeutics · Tests: Dotinurad, Placebo Taiwan Associated Genetic and Nongenetic Small Vessel Disease NCT05473637n/a1 site📍 Taipei National Taiwan University Hospital · Tests: MRI Cardiac Amyloid Deposits and Heart Dysfunction in Alzheimer's Disease NCT07154394n/a1 site📍 Essen University Hospital, Essen Decoding the Genetic Landscape of Skeletal Diseases NCT05876416n/a1 site📍 Stockholm Karolinska Institutet Cardiac Structure and Function in MPS NCT07361536n/a3 sites📍 Orange📍 Minneapolis📍 St Louis Children's Hospital of Orange County Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia NCT04159103Phase 1, Phase 235 sites📍 Los Angeles📍 Los Angeles📍 Stanford ModernaTX, Inc. · Tests: mRNA-3927 Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease NCT02437396n/a1 site📍 Minneapolis University of Minnesota 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, GM1 Gangliosidosis or GM2 Gangliosidosis NCT07054515Phase 339 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: AZ-3102, Placebo An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 NCT05295433Phase 1, Phase 212 sites📍 Los Angeles📍 Palo Alto📍 San Diego ModernaTX, Inc. · Tests: mRNA-3705
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