| Rare Diseases |
RG6013: Investigation therapy for people with hemophilia A |
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| Not yet recruiting | N/A | 0 | RoW | | Roche, Chugai | hemophilia A | | | | |
NCT06047210: Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis |
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| Completed | N/A | 14 | US | Blood Draws Data Collection | Swedish Orphan Biovitrum, Brigham and Women's Hospital, Harvard Medical School (HMS and HSDM) | Hemophagocytic Lymphohistiocytoses | 10/19 | 10/19 | | |
NCT05715749: Body Weight Support Harness System in Spinal Muscular Atrophy |
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| Active, not recruiting | N/A | 33 | US | In-home body weight support harness system, The Portable Mobility Aid for Children (PUMA) | Nationwide Children's Hospital, Novartis | Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II | 06/20 | 12/23 | | |
NCT03639844: BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study |
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| No Longer Available | N/A | | US | rivogenlecleucel, BPX-501 T cells, rimiducid, AP1903, Rimiducid for Injection, AP1903 for Injection | Bellicum Pharmaceuticals | Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism | | | | |
NCT03771391: A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU |
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| Completed | N/A | 15 | Europe | PKU Sphere, GMP, Glycomacropeptide | Vitaflo International, Ltd, Kreiskliniken Reutlingen, Johannes Gutenberg University Mainz | Phenylketonurias | 10/20 | 07/23 | | |
| Withdrawn | N/A | 1000 | Europe, RoW | | CENTOGENE GmbH Rostock | Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia, Eye Abnormalities | 02/21 | 02/21 | | |
NCT02865109: Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA) |
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| No Longer Available | N/A | | RoW | Nusinersen, ISIS 396443, BIIB058, Spinraza | Biogen | Infantile-onset Spinal Muscular Atrophy | | | | |
NCT01968655: Expanded Access to B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies |
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| No Longer Available | N/A | | US | OBI-1, B-Domain Deleted Recombinant Porcine Factor VIII | Baxalta now part of Shire | Acquired Hemophilia A | | | | |
| Recruiting | N/A | 150 | Canada | Zamplo Digital Health Platform | M.A.G.I.C. Clinic LTD, Hanalytics Solutions Inc. | Metabolic Disease, Fabry Disease, Gaucher Disease, Pompe Disease, Mitochondrial Diseases | 05/21 | 11/21 | | |
NCT04375592: Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria |
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| Completed | N/A | 10 | Europe | Phenylalanine-free protein substitute in tablet form (XPhe minis) | metaX Institut fuer Diatetik GmbH, Birmingham Children's Hospital | Phenylketonurias, Hyperphenylalaninaemia, Tetrahydrobiopterin Deficiency | 05/21 | 05/21 | | |
| Available | N/A | | NA | Pegcetacoplan | Apellis Pharmaceuticals, Inc. | C3G, IC-MPGN, C3 Glomerulopathy, C3 Glomerulonephritis, Complement 3 Glomerulopathy, Complement 3 Glomerulopathy (C3G), Complement 3 Glomerulonephritis, Dense Deposit Disease, DDD, Membranoproliferative Glomerulonephritis, Membranoproliferative Glomerulonephritis (MPGN), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | | | | |
| Active, not recruiting | N/A | 100 | RoW | | Fundación Grupo de Investigación en Cuidados Intensivos y Obstetricia, Recordati Rare Diseases | Rare Diseases, Porphyrias | 12/21 | 12/22 | | |
ZSE-DUO, NCT03563677: Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases |
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| Completed | N/A | 1379 | Europe | dual expert guidance structure | Wuerzburg University Hospital, Hannover Medical School, University Hospital, Aachen, Allianz Chronischer Seltener Erkrankungen ACHSE e.V., Goethe University, Universität Münster, St. Josef Hospital Bochum, Otto-von-Guericke University Magdeburg, University Medical Center Mainz, University Hospital Regensburg, University Hospital Tuebingen, University Hospital Ulm, IKK gesund plus, Techniker Krankenkasse, University of Wuerzburg, Universitätsklinikum Hamburg-Eppendorf, AOK Hessen, LWL-Universitätsklinikum der Ruhr-Universität Bochum | Rare Diseases, Orphan Diseases | 01/22 | 09/22 | | |
NCT04876365: A Study in Children, Teenagers and Adults With Severe Hemophilia A Who Switched From Other Factor VIII Treatments to Adynovate |
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| Completed | N/A | 153 | Canada | Non-Interventional | Takeda | Hemophilia A | 02/22 | 02/22 | | |
NCT02941783: Drug Use Investigation of Kovaltry in Hemophilia A Patients |
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| Active, not recruiting | N/A | 231 | Japan | Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973) | Bayer | Hemophilia A | 03/22 | 09/25 | | |
NCT06178393: Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States |
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| Completed | N/A | 162 | US | | Novartis Pharmaceuticals | Spinal Muscular Atrophy | 12/22 | 12/22 | | |
NCT04452513: A Prospective Clinical Study of Phenylketonuria (PKU) |
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| Completed | N/A | 32 | US | | BioMarin Pharmaceutical | Phenylketonurias | 06/22 | 06/22 | | |
| Completed | N/A | 19 | Europe | PKU sphere liquid | Vitaflo International, Ltd | Phenylketonurias | 06/22 | 02/23 | | |
| Recruiting | N/A | 5 | Europe | feasibility study | University Hospital, Bonn, Swedish Orphan Biovitrum | Sonography, Haemophilia | 07/22 | 12/22 | | |
NCT05062226: GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR |
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| Completed | N/A | 45 | Europe | PKU/TYR GMP Protein Substitute | Nutricia UK Ltd | PKU, Tyrosinemias | 08/23 | 11/23 | | |
| Active, not recruiting | N/A | 687 | Europe | CARE-FAM, WEP-CARE | Silke Wiegand-Grefe, Prof. Dr., Techniker Krankenkasse, BARMER, DAK-Gesundheit Krankenkasse, KKH Kaufmännische Krankenkasse, BKK Mobil Oil, Achse e.V., University of Ulm, Universitätsklinikum Hamburg-Eppendorf, University Hospital Schleswig-Holstein, University Medical Center Rostock, University Hospital Muenster, University Hospital, Essen, Ruhr University of Bochum, Universitätsklinikum Köln, University of Göttingen, Hannover Medical School, Evangelisches Klinikum Bethel, Charite University, Berlin, Germany, DRK Kliniken Berlin Westend, Universitätsklinikum Leipzig, University of Giessen, University Hospital Freiburg, Josefinum Augsburg, University Hospital Augsburg, University Hospital, Saarland, Jena University Hospital, Leibniz Universität, Center for Health Economics Research Hannover, aQua-Institut | Rare Diseases | 09/22 | 12/22 | | |
FAPREV-HCM, NCT04943991: Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score |
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| Active, not recruiting | N/A | 200 | Europe | blood sampling (alpha-Galactosidase & LysoGb3) | Wuerzburg University Hospital, Takeda | Fabry Disease, Fabry Disease, Cardiac Variant, Lysosomal Storage Diseases, HCM - Hypertrophic Cardiomyopathy, Anderson Fabry Disease | 10/22 | 10/24 | | |
SWITCH, NCT05041114: II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis |
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| Withdrawn | N/A | 10 | RoW | Motor Neuroprosthesis | Synchron Medical, Inc. | Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injury, Stroke, Lacunar, Stroke, Brainstem, Upper Limb Injury, Spinal Muscular Atrophy | 11/22 | 11/22 | | |
AIM-Active, NCT04845555: Association Between Individual Clotting Factor Level Monitoring and the Risk of Bleeding Whilst Physical Active Conditions |
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| Recruiting | N/A | 30 | Europe | Observation of the association between clotting factor level and risk of bleeding whilst physical activity | Prof. Dr. Dr. Thomas Hilberg, Takeda, University of Bonn | Hemophilia A | 12/22 | 12/22 | | |
| Completed | N/A | 86 | US | | American Thrombosis and Hemostasis Network, BioMarin Pharmaceutical | Hemophilia A and B, Liver Transplantation | 12/22 | 12/22 | | |
| Completed | N/A | 37 | Europe | | Nutricia Research | Phenylketonuria (PKU) | 12/22 | 04/23 | | |
NCT03006965: Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT® |
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| Completed | N/A | 50 | Europe | octocog alfa, Advate®, recombinant intravenous factor VIII, rurioctocog alfa pegol | Spanish Society of Thrombosis and Haemostasis, Takeda, Instituto de Investigación Hospital Universitario La Paz | Hemophilia A, Hemophilia, Factor VIII Deficiency | 12/22 | 12/22 | | |
NAP, NCT04671810: Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® |
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| Completed | N/A | 44 | RoW | Elizaria®, Eculizumab | AO GENERIUM | Paroxysmal Nocturnal Hemoglobinuria | 12/22 | 05/23 | | |
NCT03856203: Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase |
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| Completed | N/A | 12 | US | | Boston Children's Hospital, BioMarin Pharmaceutical | Phenylketonurias | 12/22 | 10/23 | | |
| Active, not recruiting | N/A | 19 | Europe | Glytactin, Glytactin Build 20 Flavours, Glytactin Ready To Drink (RTD) 15 Lite, Glytactin Build 10, L-AA | Ajinomoto Co., Inc., Birmingham Children's Hospital, Great Ormond Street Hospital for Children NHS Foundation Trust | Phenylketonurias | 01/25 | 04/25 | | |
| Completed | N/A | 12 | Europe | CGMP protein substitute, PKU Sphere, L-amino acid protein substitute | Vitaflo International, Ltd, Arla Foods | Phenylketonurias | 01/23 | 01/23 | | |
| Recruiting | N/A | 250 | Europe | | PD Dr. Andreas Strauß, Bayer | Osteoporosis, Haemophilia, Bone Mass | 02/23 | 03/23 | | |
| Terminated | N/A | 7 | US | | Homology Medicines, Inc | Phenylketonurias, PKU | 02/23 | 08/23 | | |
Exigency, NCT04723680: An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families |
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| Completed | N/A | 69 | Europe | Qualitative interview | Haemnet, UniQure Biopharma B.V. | Hemophilia | 02/23 | 02/23 | | |
| Completed | N/A | 71 | Europe | | Nutricia Research | Phenylketonuria | 03/23 | 05/23 | | |
NCT04689360: An Intermediate Size Expanded Access Protocol of Elamipretide |
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| Available | N/A | | NA | elamipretide, MTP-131 | Stealth BioTherapeutics Inc. | Mitochondrial Diseases, Barth Syndrome | | | | |
THIRd, NCT04584892: Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities |
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| Completed | N/A | 18 | Europe | Turoctocog alfa | Novo Nordisk A/S | Haemophilia A | 03/23 | 03/23 | | |
NCT03329365: Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS |
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| Active, not recruiting | N/A | 200 | Canada | | Lawson Health Research Institute, Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Cerebral Vein Thrombosis | 03/23 | 03/24 | | |
| Completed | N/A | 135 | Japan | ADYNOVATE, BAX 855, BAX855, Recombinant Factor VIII (FVIII) PEGylated | Takeda, Takeda | Hemophilia A | 09/23 | 09/23 | | |
MARS, NCT04551989: Mepolizumab Long-term Study to Assess Real World Safety and Effectiveness of Eosinophilic Granulomatosis With Polyangiitis (EGPA) in Japan |
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| Active, not recruiting | N/A | 120 | Japan | | GlaxoSmithKline | Churg-Strauss Syndrome | 04/23 | 04/23 | | |
HAPKIDO, NCT04953000: A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A |
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| Completed | N/A | 94 | RoW | | Takeda | Hemophilia A | 04/23 | 04/23 | | |
NCT03876301: Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants |
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| Completed | N/A | 25 | Canada, US, RoW | Standard of Care FVIII Replacement therapy | Spark Therapeutics | Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Hematologic Diseases, Hemorrhagic Disorders, Factor VIII Deficiency | 05/23 | 05/23 | | |
| Available | N/A | | NA | Iptacopan, LNP023 | Novartis Pharmaceuticals | C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH) | | | | |
NCT05409079: Schulze Muscular Dystrophy Ability Clinical Study |
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| Recruiting | N/A | 35 | US | Abilitech Assist | AbiliTech Medical Inc., Richard M. Schulze Family Foundation | Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy | 06/23 | 06/23 | | |
| Available | N/A | | NA | Danicopan, ALXN2040 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis | | | | |
NCT05284175: A Study of Orelabrutinib in Patients With AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder |
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| Not yet recruiting | N/A | 23 | RoW | Orelabrutinib | Peking Union Medical College Hospital, Beijing InnoCare Pharma Tech Co., Ltd., GCP ClinPlus Co., Ltd. | Neuromyelitis Optica Spectrum Disorder | 08/23 | 08/23 | | |
NCT06028594: Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Available | N/A | | NA | Pozelimab, REGN3918, Cemdisiran, ALN-CC5 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | | | | |
| Recruiting | N/A | 100 | Europe | Next Generation Sequencing, NGS, Whole genome Sequencing | Munich Leukemia Laboratory, Illumina, Inc. | Leukemia, Hematologic Malignancy, Rare Diseases, Refractory Leukemia, Refractory Lymphoma, Unknown Primary Tumors | 08/23 | 10/26 | | |
| Completed | N/A | 165 | Europe | Questionnaire and interview | Haemnet, BioMarin Pharmaceutical | Hemophilia | 09/23 | 09/23 | | |
| Recruiting | N/A | 30 | US | PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards | Lumos Diagnostics, Aptatek Biosciences | Phenylketonurias | 01/24 | 03/24 | | |
| Available | N/A | | NA | Pozelimab, REGN3918 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH), CD55-deficient Protein-losing Enteropathy (PLE), CD59 Deficiency | | | | |
REAL, NCT05395858: A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers |
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| Terminated | N/A | 14 | US | No intervention | Bayer | Hemophilia A | 11/23 | 11/23 | | |
ZTWINS, NCT05312814: Clinical Utility of the Addition of a SNP-based NIPT Zygosity Determination in Twin Pregnancy Management. |
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| Completed | N/A | 137 | US | | Natera, Inc. | Twin to Twin Transfusion Syndrome, Pregnancy Complications, Multiple Gestation; Maternal Care | 05/23 | 05/23 | | |
NCT04921956: Compassionate Use of Concizumab if You Have Haemophilia |
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| Available | N/A | | Europe, US | Concizumab | Novo Nordisk A/S | Congenital Haemophilia | | | | |
| Recruiting | N/A | 1000 | RoW | | AstraZeneca | Paroxysmal Nocturnal Hemoglobinuria | 11/25 | 11/25 | | |
| Active, not recruiting | N/A | 395 | US | | American Thrombosis and Hemostasis Network, Genentech, Inc. | Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor | 11/24 | 12/24 | | |
NCT05274633: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria |
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| Recruiting | N/A | 120 | Europe | Ravulizumab, Ultomiris | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria | 06/24 | 02/25 | | |
NCT05437211: A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions |
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| Completed | N/A | 24 | Europe | Virtual-reality (VR) Based Solution | Takeda | Hemophilia | 02/24 | 02/24 | | |
COMMAND, NCT05035823: Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis |
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| Active, not recruiting | N/A | 6 | US | Motor Neuroprosthesis (MNP) | Synchron Medical, Inc. | Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injuries, Stroke, Lacunar, Stroke Brainstem, Cervical Spinal Cord Injury, Spinal Muscular Atrophy, Tetraplegic; Paralysis, Quadriplegia/Tetraplegia | 08/24 | 08/24 | | |
NCT06167954: Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment |
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| Recruiting | N/A | 50 | Europe | EXPLORER | MarsiBionics, Hospital Infantil Universitario Niño Jesús, Madrid, Spain, Hospital Universitario La Paz, Hospital Universitario 12 de Octubre, Hospital General Universitario Gregorio Marañon | Cerebral Palsy, Acquired Brain Injury, Spinal Muscular Atrophy | 03/24 | 03/24 | | |
SAAVY, NCT04560933: A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A |
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| Completed | N/A | 186 | US | Blood sample collection | BioMarin Pharmaceutical | Hemophilia A | 09/23 | 10/23 | | |
| Recruiting | N/A | 800 | Europe | Patient Registry | Newcastle University, Biogen, Roche Pharma AG, Adult SMA REACH, SMA REACH UK | Spinal Muscular Atrophy, SMA | 12/23 | 12/23 | | |
NCT03399981: Tysabri Observational Cohort Study - Multiple Sclerosis (MS) Registries |
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| Completed | N/A | 80327 | US | Tysabri, Natalizumab BG00002 | Biogen | Progressive Multifocal Leukoencephalopathy | 12/23 | 12/23 | | |
B-MORE, NCT03901755: An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B |
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| Active, not recruiting | N/A | 151 | Europe, RoW | Alprolix, Eftrenonacog alfa, rFIXFc | Swedish Orphan Biovitrum, Cerner Enviza | Hemophilia B | 05/24 | 05/24 | | |
NCT04941898: A Study of TAK-660 in Surgical Procedures for People With Hemophilia A. |
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| Completed | N/A | 17 | Japan | PEGylated Recombinant Factor VIII, TAK-660, BAX855, ADYNOVATE | Takeda | Hemophilia A | 03/24 | 03/24 | | |
NCT05219487: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration |
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| Recruiting | N/A | 30 | US | Risdiplam, Evrysdi® | Bakri Elsheikh, Genentech, Inc. | Spinal Muscular Atrophy | 12/24 | 12/24 | | |
DigiNOA, NCT05109637: A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders |
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| Completed | N/A | 93 | Europe | Konectom NMD Application | Biogen | Spinal Muscular Atrophy | 07/23 | 07/23 | | |
| Active, not recruiting | N/A | 7 | Europe | PKU explore | Vitaflo International, Ltd | Phenylketonurias | 12/24 | 05/25 | | |
NCT05840055: ACT With NMOSD Patients and Caregivers Pilot Study |
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| Recruiting | N/A | 50 | US | Acceptance and Commitment Therapy | Thomas Jefferson University, Alexion Pharmaceuticals, Inc. | NMO Spectrum Disorder | 06/24 | 09/24 | | |
| Not yet recruiting | N/A | 30 | NA | Low calorie protein substitute | Nutricia UK Ltd | Phenylketonurias, Hyperphenylalaninaemia | 12/23 | 12/23 | | |
NCT05904210: Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® |
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| Completed | N/A | 17 | US | SEVENFACT® | Laboratoire français de Fractionnement et de Biotechnologies | Hemophilia | 05/24 | 05/24 | | |
| Completed | N/A | 951 | Europe, Canada, RoW | ADVATE, octocog alfa, rAHF-PFM, ADYNOVI, rurioctocog alfa pegol, rAHF-PEG | Baxalta now part of Shire, Baxalta Innovations GmbH, now part of Shire | Hemophilia A | 01/24 | 01/24 | | |
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NCT06305234: A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) |
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| Recruiting | N/A | 200 | US | Pegvaliase | BioMarin Pharmaceutical | Phenylketonuria (PKU) | 11/33 | 11/33 | | |
NCT06222697: A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A |
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| Recruiting | N/A | 600 | RoW | Damoctocog-alfa-pegol (Jivi, BAY94-9027) | Bayer | Hemophilia A, Prophylaxis of Bleeding, Treatment of Bleeding | 03/26 | 06/26 | | |
NCT03824522: Post Marketing Surveillance Study for ADYNOVATE in South Korea |
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| Completed | N/A | 341 | RoW | ADYNOVATE, PEGylated rFVIII | Takeda, Takeda Pharma Korea Co. Ltd. | Hemophilia A | 01/24 | 01/24 | | |
| Completed | N/A | 100 | US | | Mayo Clinic, Alexion Pharmaceuticals, Inc. | Neuromyelitis Optica, NMO Spectrum Disorder | 04/24 | 04/24 | | |
NCT04629274: In Vitro Study of the Biological and Immunological Activity of Imotopes® Candidates on Blood Cells of Patients With Stabilized NMO |
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| Completed | N/A | 15 | Europe | Blood sampling | Imcyse SA | Neuromyelitis Optica | 06/24 | 06/24 | | |
NCT06298448: eGPA and Local Inflammation Within the Ear, Nose and Throat Area |
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| Recruiting | N/A | 150 | Europe | laboratory experiments before and after mepolizumab treatment | University Medical Center Groningen, GlaxoSmithKline | EGPA - Eosinophilic Granulomatosis With Polyangiitis | 12/27 | 12/28 | | |
NCT06357572: Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem |
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| Completed | N/A | 6 | Europe | Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Additional coagulation tests | Enzyre B.V., Instytut Hematologii i Transfuzjologii, Warschau, Poland | Hemophilia A With Inhibitor, Hemophilia A | 03/24 | 03/24 | | |
NCT06312644: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy |
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| Not yet recruiting | N/A | 300 | NA | Ultomiris, There is no interventional drug. Ultomiris treatment is an inclusion criterion. | Alexion Pharmaceuticals, Inc. | Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), Neuromyelitis Optica Spectrum Disorder (NMOSD) | 10/33 | 10/33 | | |
NCT00315380: Longitudinal Study for Eosinophilic Granulomatosis With Polyangiitis |
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| Recruiting | N/A | 700 | Canada, US | | University of Pennsylvania, GlaxoSmithKline, AstraZeneca | Eosinophilic Granulomatosis With Polyangiitis, Churg-Strauss Syndrome | 12/28 | 12/28 | | |
| Recruiting | N/A | 300 | Europe | Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS) | University Hospital, Bonn, Swedish Orphan Biovitrum | Haemophilia A, Synovitis, Hemophilia Arthropathy, Sonography | 02/27 | 03/27 | | |
POPS or POP02, NCT04278404: Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS) |
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| Recruiting | N/A | 5000 | Canada, US | The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:, Aminocaproic acid, Amiodarone, Bosentan, Budesonide, Cefdinir, Cefepime, Ceftazidime, Clindamycin, Clobazam, Dexamethasone, Dexmedetomidine, Dextroamphetamine/Amphetamine, Fosfomycin, Furosemide, Gabapentin, Guanfacine, Hydrocortisone, Labetalol, Meropenem, Metformin, Milrinone, Nalbuphine, Nicardipine, Nifedipine, Oseltamivir, Oxycodone, Risperidone, Sertraline, Sevelamer Carbonate / Sevelamer Hydrochloride, Spironolactone, Terbutaline, Tranexamic acid, Voriconazole, Zolpidem, Azithromycin, Lopinavir/Ritonavir, Ribavirin, Tocilizumab, Anakinra, Aspirin, Canakinumab, Colchicine, Interferon, Remdesivir, Ruxolitinib, Sarilumab, Abatacept, Infliximab | Duke University, The Emmes Company, LLC, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension, Pain, Hyperphosphatemia, Primary Hyperaldosteronism, Edema, Hypokalemia, Heart Failure, Hemophilia, Menorrhagia, Insomnia, Pneumonia, Skin Infection, Arrythmia, Asthma in Children, Bronchopulmonary Dysplasia, Adrenal Insufficiency, Fibrinolysis; Hemorrhage, Attention Deficit Hyperactivity Disorder, Multisystem Inflammatory Syndrome in Children (MIS-C), Kawasaki Disease, Coagulation Disorder, Down Syndrome | 09/26 | 09/27 | | |
NCT03775954: Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise |
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| Recruiting | N/A | 200 | US | Fetal Magnetocardiogram and Neonatal Electrocardiogram | Medical College of Wisconsin, National Heart, Lung, and Blood Institute (NHLBI), University of Wisconsin, Madison, Children's Hospital and Health System Foundation, Wisconsin, Shared Medical Technology, Inc., Tristan Technologies, Inc | High Risk Pregnancy, Congenital Heart Disease, Fetal Hydrops, Twin Monochorionic Monoamniotic Placenta, Gastroschisis, Fetal Demise, Stillbirth, Fetal Arrhythmia, Long QT Syndrome, Intrauterine Fetal Death, Sudden Infant Death, Pregnancy Loss, Twin Twin Transfusion Syndrome, Birth Defect, Fetal Cardiac Anomaly, Fetal Cardiac Disorder, Fetal Death, Brugada Syndrome | 04/24 | 04/24 | | |
NCT04888702: Holter of Movement in Patients With SMA Undergoing Treatment. |
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| Active, not recruiting | N/A | 30 | Europe | Actimyo | Centre Hospitalier Universitaire de Liege, SYSNAV, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Régional de la Citadelle | Spinal Muscular Atrophy | 07/24 | 07/24 | | |
NCT05356377: Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU |
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| Enrolling by invitation | N/A | 13 | US | Pegvaliase-Pqpz | University of Missouri-Columbia, BioMarin Pharmaceutical | Phenylketonurias | 04/24 | 04/24 | | |
NN7415-7557, NCT06285071: Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors |
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| Enrolling by invitation | N/A | 23 | Japan | Concizumab, Alhemo | Novo Nordisk A/S | Haemophilia A, Haemophilia B | 04/30 | 04/30 | | |
NCT06273865: Version Testing of EnzySystem Version A for Hemophilia A |
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| Recruiting | N/A | 40 | Europe | Several assays | Radboud University Medical Center, Enzyre B.V. | Hemophilia A | 10/24 | 12/24 | | |
NCT05908656: Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight) |
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| Recruiting | N/A | 105 | US | Investigational procedure | Sanofi | Gaucher Disease | 07/24 | 07/24 | | |
NCT06369740: Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring |
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| Not yet recruiting | N/A | 102 | US | Case scenarios - questionnaire, Case scenarios - Focus groups, Use scenarios - Usability test, Use scenarios - interview | Enzyre B.V., Indiana Hemophilia & Thrombosis Center | Hemophilia A | 06/24 | 06/24 | | |
ACE SMA, NCT06419322: Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA). |
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| Recruiting | N/A | 14 | Europe | Optimized rehabilitation program | University of Oxford, Oxford Brookes University, Oxford University Hospitals NHS Trust, ACE SMA Charity, Roche Products Limited, Scholar Rock, Inc., Biohaven Therapeutics Ltd. | Spinal Muscular Atrophy | 06/26 | 12/26 | | |
NCT06461533: A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A |
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| Recruiting | N/A | 25 | Japan | Susoctocog Alfa (Genetical Recombination), OBIZUR Intravenous Injection 500, TAK-672 | Takeda | Acquired Hemophilia A | 10/29 | 10/29 | | |
aNiMatO, NCT06374264: Acceptability and Safety of MR-C-014 in Persons With Neuromyelitis Optica Spectrum Disorder |
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| Not yet recruiting | N/A | 30 | US | MR-C-014 | MedRhythms, Inc., Alexion Pharmaceuticals, Inc. | Neuromyelitis Optica Spectrum Disorder | 12/24 | 12/24 | | |
| Not yet recruiting | N/A | 128 | NA | PNH-relevant therapies | Novartis Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 05/25 | 05/25 | | |
NCT04805801: The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors |
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| Recruiting | N/A | 17 | RoW | Emicizumab subcutaneous injection, Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab test | JW Pharmaceutical | Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor | 07/24 | 02/25 | | |
NCT06191068: Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia |
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| Not yet recruiting | N/A | 288 | NA | | The Hospital for Sick Children, Roche Pharma AG | Hemophilia | 02/25 | 06/25 | | |
| Not yet recruiting | N/A | 604 | NA | | University Hospital, Clermont-Ferrand, Effik, Université d'Auvergne, AUDIPOG | Nausea and Vomiting | 10/25 | 04/26 | | |
AMAZE, NCT06398158: Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder |
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| Recruiting | N/A | 35 | US | Ravulizumab | University of Texas Southwestern Medical Center, Alexion Pharmaceuticals, Inc. | Neuromyelitis Optica | 12/26 | 06/27 | | |
| Recruiting | N/A | 10 | Europe | PKU sphere | Vitaflo International, Ltd, University College London Hospitals | Maternal Phenylketonuria | 06/25 | 06/25 | | |
| Recruiting | N/A | 29 | US | IMT therapy using the Pr02 mobile device | Duke University, Genzyme, a Sanofi Company | Late-Onset Pompe Disease, Lysosomal Disease | 08/24 | 08/24 | | |
