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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 Inherited Metabolic Disorders

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 GLP-1R Actions on Muscle and the Skeleton NCT07154719Phase 42 sites📍 Baton Rouge📍 Scarborough Pennington Biomedical Research Center · Tests: Tirzepatide, Lifestyle toolkit An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors NCT07371663Phase 1, Phase 23 sites📍 Beijing📍 Zhengzhou📍 Hangzhou Beijing Tide Pharmaceutical Co., Ltd · Tests: TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg, benmelstobart Injection Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer NCT06088030Phase 21 site📍 Guangzhou Yang Li · Tests: Arsenic trioxide Caregiving Networks Across Disease Context and the Life Course NCT05007990n/a1 site📍 Bethesda National Human Genome Research Institute (NHGRI) Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy NCT04912843Phase 2, Phase 31 site📍 Beijing Wuhan Neurophth Biotechnology Limited Company · Tests: NR082 injection, Sham Injection 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 Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria NCT03301038Phase 21 site📍 Philadelphia Children's Hospital of Philadelphia · Tests: Rifampin 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 New Markers of Glycation to Predict Gestational Diabetes Mellitus and Macrosomia. NCT06048510NA1 site📍 Bordeaux University Hospital, Bordeaux · Tests: Pregnant women 18F-DOPA II - PET Imaging Optimization NCT04706910Phase 31 site📍 Edmonton University of Alberta · Tests: 18F-DOPA, Furosemide Injection Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) NCT05071222Phase 1, Phase 21 site📍 Paris Assistance Publique - Hôpitaux de Paris · Tests: ARTEGENE drug product 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 Cerebellar TMS and Satiety in Prader-Willi Syndrome NCT05938543NA1 site📍 Belmont Brigham and Women's Hospital · Tests: Repetitive Transcranial Magnetic Stimulation (rTMS) Adiposity and Iron Requirements in Pregnancy NCT06425796NA3 sites📍 Coleraine📍 Coleraine📍 Coleraine University of Ulster · Tests: Adiposity and Iron Requirements in Pregnancy (ADIPREG) 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
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