Not applicableStudy completedNCT01632111What this trial is testingPulmonary Involvement in Patients With Fabry DiseaseWho this might be right forFabry Disease University of Zurich 110
Post-approval studies (Phase 4)Study completedNCT05134571What this trial is testingChina Post-marketing Surveillance (PMS) Study of Aldurazyme®Who this might be right forMucopolysaccharidosis I Genzyme, a Sanofi Company 12
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776What this trial is testingClinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaWho this might be right forGlycogen Storage Disease Type II Sanofi 17
Very early researchLooking for participantsNCT06458010What this trial is testingClinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)Who this might be right forFamilial Hypercholesterolemia RenJi Hospital 20
Post-approval studies (Phase 4)Study completedNCT00097890What this trial is testingReplagal Enzyme Replacement Therapy for Adults With Fabry DiseaseWho this might be right forFabry Disease National Institute of Neurological Disorders and Stroke (NINDS) 25
Large-scale testing (Phase 3)Not Yet RecruitingNCT07579910What this trial is testingIntracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)Who this might be right forMPS IIIB Spruce Biosciences 14
Post-approval studies (Phase 4)UnknownNCT04419363What this trial is testingBurosumab in Children and Adolescents With X-linked HypophosphatemiaWho this might be right forRare DiseasesX-linked Hypophosphatemia Bicetre Hospital 57
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04240821What this trial is testingTheophylline for Treatment of PseudohypoparathyroidismWho this might be right forPseudohypoparathyroidismPseudohypoparathyroidism Type 1aAlbright Hereditary Osteodystrophy Jaclyn Tamaroff 34
Testing effectiveness (Phase 2)Study completedNCT02595983What this trial is testingThe Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantWho this might be right forTransthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more Alnylam Pharmaceuticals 12
Not applicableLooking for participantsNCT06439654What this trial is testingAtlantic Lipid Lowering Treatment Optimization ProgramWho this might be right forFamilial HypercholesterolemiaLipoprotein Types--Lp System Lp(A) HyperlipoproteinemiaApolipoprotein B 100, Familial Defective+2 more Atlantic Health System 250
Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405What this trial is testingGene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIAWho this might be right forMucopolysaccharidosis Type IIIA University of Manchester 5
Large-scale testing (Phase 3)Study completedNCT01347073What this trial is testingStudy of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)Who this might be right forUrea Cycle Disorders Amgen 23
Post-approval studies (Phase 4)Study completedNCT00243542What this trial is testingPhase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD DeficiencyWho this might be right forAcne Vulgaris Allergan 64
Early research (Phase 1)Study completedNCT01603186What this trial is testingBioequivalence Study of Quetiapine Fumarate Tablets 25 mg Under Fed ConditionWho this might be right forFed IPCA Laboratories Ltd. 54
Large-scale testing (Phase 3)Study completedNCT00002070What this trial is testingPhase III Ganciclovir +/- rGM-CSF for AIDS-Related CMV RetinitisWho this might be right forCytomegalovirus RetinitisHIV Infections Schering-Plough
Testing effectiveness (Phase 2)Study completedNCT07010575What this trial is testingPatient Preference Study: Standard of Care Versus Once-daily Trientine TetrahydrochlorideWho this might be right forWilson's Disease Orphalan 10
Large-scale testing (Phase 3)Study completedNCT02585895What this trial is testingEvolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study EnrollmentWho this might be right forHypercholesterolemia Amgen 39
Testing effectiveness (Phase 2)Study completedNCT02489344What this trial is testingEvaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry DiseaseWho this might be right forFabry Disease Genzyme, a Sanofi Company 8
Not applicableStudy completedNCT04281537What this trial is testingDescribe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.Who this might be right forFabry Disease Amicus Therapeutics 82
Post-approval studies (Phase 4)UnknownNCT02525172What this trial is testingImmune Modulation Therapy for Pompe DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 8