| Rare Diseases |
Hemophilia-6: Mononine is indicated for the prevention and control of bleeding in Factor IX deficiency, also known as Hemophilia B |
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| Approved for marketing | 4 | 0 | RestOfWorld | Mononine (human plasma derived coagulation factor IX) | CSL Behring | Factor IX | | | | |
2020-003256-32: A Study to Evaluate the Impact of Emicizumab on Health-Related Quality Of Life, Physical Activity, and Joint Health in Patients with Severe Hemophilia A without Factor VIII Inhibitors |
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| Ongoing | 4 | 50 | Europe | Hemlibra/Emicizumab, Ro 553-4262/F03-01, Solution for injection, Hemlibra | F. Hoffmann-La Roche Ltd, F. Hoffman-La Roche Ltd. | Severe hemophilia A without factor VIII inhibitors, Hemophilia A is deficiency in clotting factor VIII, which causes increased bleeding. Severe hemophilia A can occur after injury or spontaneously, often in joints and muscles., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2006-002635-24: A STUDY OF IMMUNOLOGIC SAFETY FOR ALPHANATEŇ IN PREVIOUSLY TREATED PATIENTS DIAGNOSED WITH SEVERE HEMOPHILIA A |
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| Ongoing | 4 | 50 | Europe | Alphanate®, Concentrate for solution for infusion, Alphanate® | Grifols Biological Inc | severe hemophilia A | | | | |
ChiCTR-TRC-10001093: Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial |
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| Completed | 4 | 40 | | Rat nerve growth factor injection for six monthes, ;Rat nerve growth factor injection for six monthes, | PLA General Hospital; Xia Men Bioway Biotech Co., LTD, Xia Men Bioway Biotech Co., LTD | children with spinal muscular atrophy | | | | |
2010-021343-41: Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin |
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| Ongoing | 4 | 20 | Europe | Soluble tablet, Kuvan | University Medical Center Groningen, Merck KGaA | Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the disease will result in high concentrations of phenylalanine (Phe) in blood and tissues, likely resulting in severe mental retardation and behavioural problems. Treatment focusus on the restriction of dietary phenylalanine intake with supplementation of a synthetic phenylalaninefree amino acid mixture., Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder. Untreated, PKU can lead to mental retardation and behavioural problems., Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
2015-001807-29: Pharmacokinetic study of rituximab induction regimen in ANCA-associated vasculitis |
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| Not yet recruiting | 4 | 20 | Europe | Rituximab, Concentrate for solution for injection, Rituximab | CHU de Saint-Etienne, THERADIAG | Granulomatosis with polyangiitis Microscopic polyangiitis granulomatose avec polyangéite (GPA) et la polyangéite microscopique (PAM), systemic vasculitis, Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01] | | | | |
2015-001090-40: Effects of hydrocortisone injection into deltoid and thigh muscles |
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| Ongoing | 4 | 8 | Europe | Hydrocortisone Efcortesol & Solu-Cortef, Injection, Efcortesol | The London Clinic | Addison's Disease, Adrenal Insufficiency, Body processes [G] - Metabolic Phenomena [G03] | | | | |
2005-001768-30: Comparison of 2 and 4 doses hydrocortison substitution with regard to plasma concentration and quality of life. |
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| Ongoing | 4 | 12 | Europe | Hydrocortisone, | EM-kliniken | We aim to study patients with cortisol deficiency caused by primary adrenal insufficiency (Addison’s disease of autoimmune origin). | | | | |
2006-005216-27: Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. |
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| Ongoing | 4 | 1 | Europe | Aldurazyme | HUS, Hospital for Children and Adolescents | Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respiratory dysfunction, and in severe fore, mental retardation | | | | |
2007-001375-11`ACTRN12610000825055: Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme |
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| Ongoing | 4 | 100 | Europe | Myozyme, MYOZYME®, Myozyme, Myozyme | Carolina Daniel de Lima-Alvarez, Foundation for Research Support of Sao Paulo, National Council for Scientific and Technological Development | head movements in pre-term infants, head movements in fullterm infants | | | | |
2007-005647-14: Gamma Delta T Cells and their role in the acute phase reaction to intravenous bisphosphonates |
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| Ongoing | 4 | 120 | Europe | Bonviva, Aclasta, Bonviva, Aclasta | Cardiff University | Patients with osteoporosis or Paget\'s disease of bone | | | | |
2008-006465-85: Pharmacokinetic evaluation of a new formulation of recombinant factor IX (BeneFIX) in the italian population with severe or moderate B haemophilia previously treated. |
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| Ongoing | 4 | 50 | Europe | BENEFIX, BENEFIX | AZIENDA OSPEDALIERA UNIVERSITARIA CAREGGI | Patients with severe or moderate B Haemophilia, previously treated with rFIX. | | | | |
2009-017060-17: STIMULATE study: Statins influence on Minrin upregulation of von Willebrand factor and factor VIII |
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| Ongoing | 4 | 20 | Europe | simvastatin, Desmopressine, simvastatine, Minrin, simvastatine, Minrin | Academic Medical Center | Type 1 von Willebrand disease and mild hemophilia A | | | | |
2009-016029-32: MECHANISM OF ACTION AND EFFICACY OF AN ALTERNATIVE IMMUNOTOLERANCE REGIMEN BASED ON MODULATION OF IMMUNE RESPONSES: A NEW CHANCE FOR HAEMOPHILIA PATIENTS WITH REFRACTORY INHIBITORS ? |
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| Ongoing | 4 | 8 | Europe | Oral solution, Capsule, soft, Tablet, Powder for solution for injection | STRUTTURA SEMPLICE GESTIONE-ORG.-FUNZ. HUB EMOFILIA | Haemophilic patients, with high responding inhibitor (>5 BU/ml), in whom previous ITI failed and who undergo the second line ITI protocol | | | | |
2009-017369-47: An in-vitro observational pilot study of the role of gamma delta T cells in the acute phase reaction to intravenous bisphosphonate; in blood samples donated by patients receiving intravenous bisphosphonate treatment for Paget’s disease or Osteoporosis final Version 1.6 |
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| Ongoing | 4 | 20 | Europe | Zoledronate (Aclasta), | Research and Development Office Cardiff and Vale NHS Trust | Osteoporosis and Paget\'s disease | | | | |
2013-002686-19: Inhibitor study in patient with hemophilia never treated in the past Studio degli inibitori in pazienti emofilici mai trattati in precedenza |
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| Ongoing | 4 | 3 | Europe | Advate, no info available, Advate, Advate | Fondazione IRCCS Ca\' Granda Ospedale Maggiore Policlinico di Milano, Lund University, Clinical Coagulation Research Unit, Skane University Hospital, Baxter | Infants or children with severe haemophilia A previously untreated with factor concentrates. Bambini con grave emofilia A non trattati in precedenza con fattori antiemofilici. | | | | |
2014-005435-14: Combination treatment with DDAVP and factor VIII clotting factor concentrates in patients with mild haemophilia A. Combinatiebehandeling van DDAVP en factor VIII-concentraten in patiënten met milde hemofilie A. |
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| Ongoing | 4 | 60 | Europe | Solution for injection/infusion, Powder and solvent for solution for injection, Minrin, Octostim, Advate, Kogenate Bayer, Helixate NexGen, ReFacto AF, Aafact, Octanate, NovoEight | Erasmus University Medical Center, ZonMw, Ferring BV, Erasmus University Medical Centre | Mild hemophilia A patients with a FVIII plasma levels above 0.05 IU/mL. Milde hemofilie A-patiënten, A clotting disorder in which clotting factor VIII is deficient, which can cause bleeding. In this study we will look at patients with the mild form. Een stollingsstoornis waarin het gehalte van stollingsfactor VIII is verlaagd. Hierdoor kunnen bloedingen veroorzaakt worden. In deze studie kijken we naar patiënten met een milde vorm., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2016-001875-57: Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions Desmopressine en FVIII-concentraat combinatiebehandeling in niet-ernstige hemofilie A-patiënten die een kleine ingreep ondergaan |
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| Ongoing | 4 | 70 | Europe | Solution for injection/infusion, Solution for injection, Minrin, Octostim | Erasmus University Medical Center Rotterdam, Erasmus University Medical Center Rotterdam, Innovatiefonds zorgverzekeraars | Hemophilia A Hemofilie A, Hemophilia A, a bleeding disorder caused by clotting fcator VIII deficiency, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
ChiCTR-OPB-16007730: Mycophenolate Mofetil Treatment of Neuromyelitis Optica in Chinese Patients (MONICA) |
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| Recruiting | 4 | 100 | | Mycophenolate Mofetil | The third affiliated hospital, Sun Yat-sen university; The third affiliated hospital, Sun Yat-sen university, Manufacturer of sponsorship | Neuromyelitis optica | | | | |
2017-003902-42: Relationship between coagulation factor IX (FIX) genotype and plasma persistance (pharmacokinetics) of recombaint FIX (Nonacog alfa), administered for replacement therapy, in hemophilia B patients Relazioni fra genotipo (mutazione) del fattore IX (FIX) della coagulazione e permanenza plasmatica (farmacocinetica) del FIX ricombinante (Nonacog alfa), usato per terapia sostitutiva, in pazienti affetti da emofilia B |
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| Ongoing | 4 | 60 | Europe | Nonacog alfa, Powder and solvent for solution for injection, BeneFIX 250 UI powder and solvent for solution for injection, BeneFIX 500 UI powder and solvent for solution for injection, BeneFIX 1000 UI powder and solvent for solution for injection, BeneFIX 1500 UI powder and solvent for solution for injection, BeneFIX 2000 UI powder and solvent for solution for injection, BeneFIX 3000 UI powder and solvent for solution for injection | Consorzio Futuro in Ricerca, Pfizer s.r.l. | Deficiency of coagulation factor IX (FIX; Hemophilia B); Hemophilia B patients treated with recombinant FIX (BeneFIX). Carenza di fattore IX (FIX) della coagulazione (emofilia B); pazienti emofilici B trattati con FIX ricombinante (BeneFIX)., Patients affected by deficiency of coagulation factor IX (FIX; Hemophilia) and treated with substitutive therapy during which the missing factor, produced by recombinant DNA technology, is infused. Pazienti affetti da carenza di fattore IX (FIX) della coagulazione (emofilia B) trattati con terapia sostitutiva per infusione del fattore mancante, prodotto mediante tecnologie del DNA ricombinante., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2016-002510-50: A low-interventional Multicentre Post-Authorisation Safety Study for Voncento/Biostate/Aleviate for Routine Prophylaxis, Treatment of Bleeding Events and/or Surgery in Male Subjects with Haemophilia A |
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| Ongoing | 4 | 100 | Europe | Voncento, Powder and solution for solution for injection, Voncento | CSL Behring GmbH, CSL Behring GmbH | Haemophilia A, Haemophilia A is a hereditary bleeding disorder due to a deficiency of functional plasma coagulation FVIII, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2017-005197-19: Study to evaluate if switching from plasma-derived factor VIII products to recombinant factor VIII products lead to an increased inhibitor development in patients with hemophilia A Studio per valutare se il passaggio da prodotti plasma derivati di fattore VIII a prodotti ricombinanti di fattore VIII non provochi un aumento di inibitore contro il fattore VIII in pazienti con emofilia A |
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| Ongoing | 4 | 200 | Europe | Fattore VIII plasma-derivato, Fattore VIII ricombinante, pdFVIII, rFVIII, Concentrate for solution for infusion, KLOTT - 500 UI/10 ML POLV E SOLVENTE PER SOLUZIONE PER INFUSIONE 1 FLAC.NO POLVERE + 1 FLAC.NO SOLVENTE DA 10 ML + SET PER LA RICOSTITUZIONE/SOMMINISTRAZION, ADVATE - 500 UI POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE 1 FLACONCINO + 1 FLACONCINO 5 ML USO ENDOVENOSO | FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE POLICLINICO | Severe hemophilia A Emofilia A grave, Severe hemophilia A Emofilia A grave, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
NCT04419363: Burosumab in Children and Adolescents With X-linked Hypophosphatemia |
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| Recruiting | 4 | 57 | Europe | Burosumab Injection | Bicetre Hospital | Rare Diseases, X-linked Hypophosphatemia | 03/19 | 09/22 | | |
2019-001202-14: Effect of an antidepressant on chest pain in patients with achalasia |
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| Not yet recruiting | 4 | 68 | Europe | Citalopram, Tablet, Citalopram | Amsterdam UMC, None | Achalasia is a motility disorder of the esophagus. Disappearance of myenteric neurons in the esophageal wall leads to failure of relaxation of the lower esophageal sphincter (LES) and impaired peristalsis. Symptoms of achalasia include dysphagia, regurgitation, chest pain and weight loss due to the stasis of food and liquids in the esophagus. Treatments effectively diminish the symptoms dysphagia and regurgitation, however have little effect on the occurrence of chest pain., Achalasia is a motility disorder of the esophagus. Symptoms of achalasia include dysphagia, regurgitation, chest pain and weight loss due to the stasis of food and liquids in the esophagus., Diseases [C] - Symptoms and general pathology [C23] | | | | |
2019-001453-10: A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Un estudio de baja intervención que documenta la eficacia, la calidad de vida relacionada con la salud y la seguridad del tratamiento se referencia con eculizumab o el tratamiento con ravulizumab en pacientes con hemoglobinuria nocturna paroxística |
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| Ongoing | 4 | 200 | Europe | ECULIZUMAB, Ultomiris, Concentrate for solution for infusion, Soliris 300, ULTOMIRIS | Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd, F. Hoffman-La Roche Ltd., Chugai Pharmaceutical Co. Ltd | Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label Hemoglobinuria paroxística nocturna (HPN) que se trata con eculizumab o ravulizumab según la etiqueta local, PNH is a rare, acquired life-threatening disease, characterized by presence of impaired red blood cells that are easily destroyed, leading to hemoglobin in the urine and anemia as main manifestations HPN es enfermedad rara, adqui q pone en peligro la vida, por la presencia de glóbu rojos deteriorados q se destruyen fácil, lo q lleva a la hemoglobina en orina y anemia como manifestaciones ppales, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
| Not yet recruiting | 4 | 50 | Europe | | Spark Therapeutics, Inc, Spark Therapeutics, Inc | Hemophilia A, Hemophilia A, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
ChiCTR-OPC-15007481: L-DEP regimen as a salvage therapy for refractory Epstein Barr virus-induced hemophagocytic lymphohistiocytosis |
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| Recruiting | 4 | 120 | | L－DEP regimen | Beijing Friendship Hospital, Capital Medical University; Beijing Friendship Hospital, Capital Medical University, subject and self-raised funds | hemophagocytic Lymphohistiocytosis | | | | |
| Active, not recruiting | 4 | 140 | Japan | Rituximab, Rituxan, Glucocorticoids, Predonine, Prednisolone | Chiba University | Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Microscopic Polyangiitis, Wegener Granulomatosis | 12/19 | 06/21 | | |
| Not yet recruiting | 4 | 76 | Europe | Film-coated tablet, Plaquenil - Hydroxychloroquine 200mg Film coated tablets | Guy's and St. Thomas' NHS Foundation Trust, Medical Research Council | The term ANCA-associated vasculitis (AAV) describes a subset of primary small vessel vasculitides characterized by the presence of anti-neutrophil cytoplasmic antibodies (ANCA): Granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA) and Eosinophilic Granulomatosis with Polyangiitis (EGPA). AAV are serious multisystem autoimmune disorders that can affect any organ in the body and commonly involve the ear-nose-throat, lungs, kidneys, eyes and joints, ANCA vasculitis is the name of a group of autoimmune conditions characterized by the inflammation of the blood vessels caused by the immune system mistakenly attacking them., Body processes [G] - Immune system processes [G12] | | | | |
2019-003427-38: An international investigator trial to capture different approaches in the treatment of Hemophilia A Patients With FVIII Inhibitors |
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| Ongoing | 4 | 120 | Europe, RoW | Octanate, Wilate, Nuwiq, Hemlibra, NovoSeven, Powder and solvent for solution for injection, Solution for injection, Octanate, Wilate, Nuwiq, Hemlibra, FEIBA NF 500 E/1000 E, NovoSeven | HZRM – Hämophilie-Zentrum Rhein Main GmbH, Octapharma AG | Inhibitor-Positive patients with Haemophilia A, Haemophilia A in patients who have developed inhibitors to any FVIII product., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2019-003440-74: Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab |
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| Not yet recruiting | 4 | 20 | Europe | ULTOMIRIS, ALXN1210, Concentrate for solution for infusion, ULTOMIRIS | Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals, Inc. | Paroxysmal nocturnal hemoglobinuria (PNH), Paroxysmal nocturnal hemoglobinuria (PNH), Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
| Not yet recruiting | 4 | 30 | Europe | Betametason Evolan 0,5 mg tablet, Betametason Evolan 0,5mg tablet, Tablet, Betametason Evolan 0,5mg | Sahlgrenska University Hospital, Gothenburg, Swedish government funding ALF-agreement, Swedish research counsil | Adrenal insufficiency e.g. Addison´s disease, Patients with Addison´s disease completely lack production of glucocorticoids (steroidhormones),which are essential for life as they play an important role for a wide range of physiological functions., Diseases [C] - Hormonal diseases [C19] | | | | |
ChiCTR2200056362: Extensively Hydrolyzed Formulas for Perioperative Nutritional Support in Children with Hirschsprung’s Disease: A randomized clinical trial |
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| Completed | 4 | 130 | | extensively hydrolyzed formula ;conventional diet | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, National Natural Science Foundation of China (81873848) | Hirschsprung’s Disease | | | | |
2021-001174-29: Effectiveness of methylphenidate for treating ADHD in adultswith PKU |
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| Not yet recruiting | 4 | 5 | Europe | Capsule, Medikinet | Amsterdam UMC, Amsterdam UMC | Phenylketonuria, ADHD Fenylketonurie, ADHD, PKU, ADHD PKU, ADHD, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| Recruiting | 4 | 48 | Europe | Paracetamol 120Mg/5mL Oral Suspension | Mette Cathrine Oerngreen, Elsass Foundation | SMA II, Cerebral Palsy | 12/24 | 12/24 | | |
NCT04108260: The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients |
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| Recruiting | 4 | 5 | RoW | Albutrepenonacog Alfa 1 UNT [IDELVION] | National Taiwan University Hospital | Hemophilia | 11/21 | 04/22 | | |
| Recruiting | 4 | 30 | Europe | Betamethasone | Göteborg University | Addison Disease | 11/21 | 12/21 | | |
NCT04784988: Intensive Replacement Treatment in Haemophilia Patients With Synovitis |
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| Completed | 4 | 64 | Europe | EHL-FVIII, FVIII concentrate | Federico II University | Hemophilia A | 03/23 | 03/24 | | |
ChiCTR2000035067: A multicentre, prospective, single arm study of bortezomib combined with glucocorticoid in the salvage treatment of relapsed / refractory acquired hemophilia A (AHA) |
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| Recruiting | 4 | 20 | | bortezomib combined with glucocorticoid | Institute of Hematology& Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology& Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, None | Relapsed / refractory acquired hemophilia A (AHA) | | | | |
| Recruiting | 4 | 20 | RoW | Mycophenolate mofetil combined with oral prednisone | Tongji Hospital | Rosai-Dorfman Disease, Langerhans Cell Histiocytosis | 06/22 | 06/22 | | |
| Completed | 4 | 90 | Europe | Phenylalanine, Placebo | University Hospital Inselspital, Berne, University of Zurich | Phenylketonuria | 06/22 | 06/22 | | |
ChiCTR1900024965: A randomized single-blind controlled clinical study: the efficacy and safety of Hiporfin-PDT in the treatment of Extramammary Paget's disease |
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| Recruiting | 4 | 20 | | Hiporfin-PDT ;ALA-PDT | Shanghai Skin Disease Hospital; Chongqing Maile Biopharmaceutics Co., Ltd., Chongqing Maile Biopharmaceutics Co., Ltd | Extramammary Paget's disease | | | | |
RELOQ, NCT04728217: Health Related Quality of Life of Youth and Young Adults With Haemophilia A |
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| Completed | 4 | 30 | RoW | Efmoroctocog Alfa Injection [Eloctate] | The League of Clinical Research, Russia | Hemophilia A | 02/23 | 02/23 | | |
2019-005007-40: Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen. Studie van het natuurlijk ziekteverloop bij volwassen patiënten met spinale spieratrofie (SMA) en de rol van biomerkers bij SMA patiënten die met nusinersen behandeld worden. |
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| Not yet recruiting | 4 | 19 | Europe | Solution for injection, Spinraza | University Hospitals Leuven, Biogen | Adult patients with spinal muscular atrophy (SMA) type 2, type 3, or type 4, spinal muscular atrophy (SMA), Diseases [C] - Nervous System Diseases [C10] | | | | |
ChiCTR1900025744: Clinical effectiveness and safety of Rituximab (Henlius) for optic neuritis secondary to neuromyelitis optica spectrum disorder: a pilot clinical trial |
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| Recruiting | 4 | 60 | | Intravenous 200mg Rituximab, 1/week*2, if the proportion of CD19+ B lymphocytes in peripheral blood is above 1% then repeat administration (200mg) ;Oral azathioprine (2.5-3 mg/kg bodyweight/day) or mycophenolate mofetil (750～3000mg/day) | The Ophthalmology Department of the Chinese Peoples Liberation Army Hospital; The General Hospital of Chinese Peoples Liberation Army, funded by the National Natural Science Fundation of China (NO.81870662) | neuromyelitis optica spectrum disorder （NMOSD） | | | | |
ACTRN12621000415808: A randomized study investigating whether taking a steroid medication (dexamethasone) for three days reduces the risk of an acute phase response reaction |
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| Completed | 4 | 60 | | | Bone & Joint Research Group, University of Auckland , Health Research Council of New Zealand | osteoporosis
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| Recruiting | 4 | 95 | Europe | Emicizumab - PK-guided dose reduction, Hemlibra, Emicizumab - Dosis continuation group, Emicizumab - Dose adjustment group | Kathelijn Fischer, Erasmus Medical Center, Amsterdam University Medical Center, Leiden University Medical Center, Radboud University Medical Center, Maastricht University Medical Center, University Medical Center Groningen, HagaZiekenhuis, Dutch Society of Haemophilia Patients | Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor, Hemophilia A, Severe, Adolescent, Child, Adult | 03/26 | 08/26 | | |
NCT04205175: A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab |
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| Active, not recruiting | 4 | 20 | US | Feiba | Children's Hospital Los Angeles, Takeda | Hemophilia A With Inhibitor | 10/22 | 10/22 | | |
ChiCTR2000041208: A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy |
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| Recruiting | 4 | 90 | | salbutamol sulfate tablets ;placebo | The Children's Hospital, Zhejiang University School of Medicine; Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd., Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd. | spinal muscular atrophy | | | | |
NCT05896605: Monitoring of Azathioprine Metabolite Concentrations and Cytokine Levels in Neuromyelitis Optica Spectrum Disorder |
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| Completed | 4 | 63 | RoW | Azathioprine, Azathioprine Tablets | First Affiliated Hospital of Guangxi Medical University | NMO Spectrum Disorder, Azathioprine Adverse Reaction | 12/22 | 04/23 | | |
ChiCTR2000035002: The pharmacokinetics reasearch of busulfan used in hematopoietic stem cell transplantation in pediartric patints, a prospective and open clinical reasearch |
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| Recruiting | 4 | 60 | | busulfan | Pediatric Hospital affiliated to Fudan University; Pediatric Hospital affiliated to Fudan University, Government, Hospital | Rare diseases in children | | | | |
ChiCTR2100050945: A prospective self-control study of glucocorticoids in adult patients with paroxysmal nocturnal hemoglobinuria |
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| Recruiting | 4 | 40 | | Glucocorticoid | Jiangsu Province Hospital; Jiangsu Province Hospital, self-funding | paroxysmal nocturnal hemoglobinuria | | | | |
NCT05728528: Impact of Moderate Intensity Physical Activities on PK-guided EHL FVIII Concentrates Prophylaxis Severe HA Patients |
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| Completed | 4 | 13 | RoW | combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis, pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis alone | Chulalongkorn University | Hemophilia | 12/22 | 12/22 | | |
| Completed | 4 | 34 | Europe | Kuvan®, Sapropterin dihydrochloride | BioMarin Pharmaceutical | Phenylketonuria | 01/23 | 01/23 | | |
| Completed | 4 | 50 | RoW | ADVATE | Baxalta now part of Shire, Takeda Development Center Americas, Inc. | Hemophilia A | 02/23 | 02/23 | | |
NCT05350020: The Effects of Two Brands of Hydrocortisone Injected Intramuscularly Into Deltoid and Thigh Muscles |
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| Recruiting | 4 | 8 | Europe | Solu-Cortef 100 MG Injection, Hydrocortisone 100mg/ml | The London Clinic | Addison's Disease | 03/23 | 08/23 | | |
ChiCTR2200059494: Pharmacokinetics of human coagulation factor IX in children with hemophilia B in China |
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| Not yet recruiting | 4 | | | | Beijing Children’s Hospital, Capital Medical University; Beijing Children’s Hospital, Capital Medical University, Enterprise | hemophilia B | | | | |
ChiCTR2100045463: Efficacy and safety of Adalimumab in refractory intestinal Behcet’s disease: A single-center, interventional, open-label, single-arm study |
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| Recruiting | 4 | 30 | | adalimumab | The First Affiliated Hospital, College of Medicine, Zhejiang University; The First Affiliated Hospital, College of Medicine, Zhejiang University, none | intestinal Beh?et's disease | | | | |
2022-002611-29: DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD) Denosumab voor de behandeling van Fibreuze Dysplasie/McCune-Albright syndroom in volwassenen (de DeFiD studie) |
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| Not yet recruiting | 4 | 82 | Europe | Injection, Denosumab (Xgeva), Natrium Chloride 0.9% | Leiden University Medical Center, Leiden University Medical Center | Fibrous Dysplasia/ McCune Albright Syndrome Fibreuze Dysplasie/McCune Albright Syndroom, Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease, consisting of the replacement of normal bone tissue with fibrous tissue. Fibreuze dysplasie is een zeldzame genetische aandoening ve, waarbijgezond bot wordt vervangen door fibreus weefsel., Diseases [C] - Hormonal diseases [C19] | | | | |
ChiCTR2100047583: Clinical study of recombinant human thrombopoietin (rhTPO) in platelet engraftment after umbilical cord blood transplantation in lysosomal storage diseases |
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| Recruiting | 4 | 20 | | Subcutaneous injection of recombinant human thrombopoietin (300U/kg) | Children's Hospital, Capital Institute of Pediatrics; Children's Hospital, Capital Institute of Pediatrics, self-funded | lysosomal storage diseases | | | | |
| Not yet recruiting | 4 | 50 | RoW | Advate, Powder and solvent for solution for injection, Advate | Takeda Biopharmaceuticals India Pvt. Ltd., Takeda Biopharmaceuticals India Pvt. Ltd. | Hemophilia A, Hemophilia A, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
OFELIA, NCT05073133: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) |
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| Completed | 4 | 16 | RoW | OAV101, AVXS-101, Zolgensma | Novartis Pharmaceuticals | Muscular Atrophy, Spinal | 08/23 | 08/23 | | |
| Terminated | 4 | 4 | Europe, Canada, Japan, US, RoW | Satralizumab 120 mg | Hoffmann-La Roche, Chugai Pharmaceutical Co. | Neuromyelitis Optica Spectrum Disorder, NMOSD | 10/23 | 10/23 | | |
| Recruiting | 4 | 80 | US | Atropine challenge, Esophageal muscle biopsy | Emory University, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Achalasia | 09/24 | 12/24 | | |
ChiCTR2100043013: Efficacy difference between low dose rituximab and mycophenolate mofetil in preventing recurrence of NMOSD |
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| Not yet recruiting | 4 | 60 | | low dose rituximab ;mycophenolate mofetil | Shandong University Qilu Hospital; Shandong University Qilu Hospital, self-finance | Neuromyelitis optica spectrum diseases | | | | |
ChiCTR2100052152: Real-World evidence of SCT800 for prophylaxis treatment based on health resource among patients with moderate or servere hemophilia A in China: a prospective observational study |
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| Not yet recruiting | 4 | 300 | | None | Hematology Hospital of Chinese Academy of Medical Sciences; Shenzhou Cell Engineering Co., Ltd., self-raised | hemophilia A | | | | |
ChiCTR2200057620: Study on dose-effect relationship and intelligent model of muscle relaxant in infants |
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| Recruiting | 4 | | | | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, self-raised | Hirschsprung's disease | | | | |
LEAPS, ChiCTR2000037821: Combining pharmacokinetics and comprehensive evaluation system to guide the prophylaxis of Chinese pediatric hemophilia A patients with Kovaltry |
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| Not yet recruiting | 4 | 60 | | adjust dosing regimens under the | Beijing Children’s Hospital, Capital Medical University; Beijing Children's Hospital, Bayer company | Hemophilia A | | | | |
ChiCTR2200056637: A multicenter, open and exploratory clinical study of pomadomide combined with dexamethasone in the treatment of newly diagnosed POEMS syndrome |
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| Recruiting | 4 | 30 | | Pomadomide combined with dexamethasone in the treatment of patients with newly diagnosed POEMS syndrome | Qingdao Municipal Hospital; Qingdao Municipal Hospital, Nothing | Multiple myeloma | | | | |
| Not yet recruiting | 4 | 60 | | Dexamethasone + tofacitinib ;Tofacitinib + etoposide + dexamethasone ;High-dose decitabine ;gemcitabine + mitoxantrone + oxaliplatin + dexamethasone | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Top Young Talents Program of Hubei Province 2021 | hemophagocytic lymphohistiocytosis | | | | |
| Recruiting | 4 | 20 | US | Emicizumab, Hemlibra | Indiana Hemophilia &Thrombosis Center, Inc., Genentech, Inc. | Factor VIII Deficiency, Congenital | 07/25 | 07/27 | | |
2023-000864-67: Safety and efficacy of intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) |
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| Not yet recruiting | 4 | 16 | RoW | Solution for injection, Zolgensma | NOVARTIS BIOCIÊNCIAS S.A, Novartis Pharma AG | Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
NCT06212245: A Clinical Research Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorders |
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| Not yet recruiting | 4 | 33 | RoW | Inebilizumab | Hansoh BioMedical R&D Company, Horizon Therapeutics Ireland DAC | Neuromyelitis Optica Spectrum Disorders | 12/24 | 06/25 | | |
2021-003537-11: A Post Approval Commitment study to evaluate the efficacy, safety, and pharmacokinetics of KOVALTRY in Chinese children, adolescents/adults with severe hemophilia A. |
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| Not yet recruiting | 4 | 48 | RoW | Kovaltry, BAY 81-8973, Powder for solution for injection/infusion, Kovaltry | Bayer Healthcare Company Limited, Bayer Healthcare Company Limited | KOVALTRY, indicated for use in adults and children with hemophilia A (congenital Factor VIIIdeficiency) for:- Routine prophylaxis to reduce the frequency of bleeding episodes- On-demand treatment and control of bleeding episodes- Perioperative management of bleeding, Treatment of hemophilia A patients (lacking of native FVIII), Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
| Recruiting | 4 | 30 | Europe, RoW | Blood tests, Inebilizumab, UPLIZNA | Amgen | Neuromyelitis Optica Spectrum Disorder | 06/28 | 06/28 | | |
NCT03654066: Botox or Botox With Esophageal Dilation in Patients With Achalasia |
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| Enrolling by invitation | 4 | 50 | US | Botulinum toxin type A, Botox, Endoscope balloon dilator, Patient reported outcomes, Barium esophagram | Vanderbilt University Medical Center | Achalasia | 06/25 | 06/25 | | |
NCT04944524: Comparison of Tofacitinib and Methotrexate in the Maintained Treatment of GPA |
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| Recruiting | 4 | 66 | RoW | Tofacitinib, Tof, Methotrexate, MTX | Shanghai Zhongshan Hospital | Granulomatosis With Polyangiitis | 07/24 | 07/24 | | |
| Recruiting | 4 | 25 | US | Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG)., Jivi, BAY94-9027 | Bayer | Hemophilia A, Prophylaxis of Bleeding | 08/24 | 09/24 | | |
| Recruiting | 4 | 76 | Europe | Hydroxychloroquine, Plaquenil, Placebo, Control | Guy's and St Thomas' NHS Foundation Trust, Medical Research Council | ANCA Associated Vasculitis, Microscopic Polyangiitis, Churg-Strauss Syndrome, Wegener Granulomatosis | 09/24 | 03/25 | | |
| Active, not recruiting | 4 | 52 | US | FVIII, Emicizumab | Children's Hospital Los Angeles, Grifols Biologicals, LLC | Hemophilia A, Immune Tolerance | 09/24 | 04/25 | | |
ChiCTR2100048364: A single-arm, prospective, and multicenter study of rituximab combined with lenalidomide in Castleman disease |
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| Not yet recruiting | 4 | 20 | | Rituximab+lenalidomide | Department of Hematology, Jiangsu Provincial People's Hospital; Jiangsu Provincial People's Hospital, National Natural Science Foundation of China | Castleman disease | | | | |
| Recruiting | 4 | 55 | US | coagulation factor VIIa [recombinant]-jncw, SEVENFACT | American Thrombosis and Hemostasis Network, LFB USA, Inc. | Hemophilia A With Inhibitor, Hemophilia B With Inhibitor | 11/24 | 06/25 | | |
NCT05491304: Cytokine Guided Risk Stratification and Treatment in Pediatric Hemophagocytic Lymphohistiocytosis |
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| Recruiting | 4 | 400 | RoW | Dexamethasone, Steroid, Corticosteroid, Etoposide, VP16, Ruxolitinib, JAK1/2 inhibitor | The Children's Hospital of Zhejiang University School of Medicine, The Second Hospital of Anhui Medical University, Union hospital of Fujian Medical University, Children's Hospital of Fudan University, Zunyi Medical College, Hunan Provincial People's Hospital, Tongji Hospital, Children's Hospital of Nanjing Medical University, The Affiliated Hospital of Qingdao University, Qilu Hospital of Shandong University, Shanghai Children's Medical Center, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Children's Hospital, Shenzhen Children's Hospital, West China Second University Hospital, Children's Hospital Of Soochow University, Second Affiliated Hospital of Wenzhou Medical University, Wuhan Children's Hospital, Institute of Hematology & Blood Diseases Hospital, The Third Xiangya Hospital of Central South University, First Affiliated Hospital, Sun Yat-Sen University | Hemophagocytic Lymphohistiocytoses, Cytokine Storm | 12/24 | 12/25 | | |
ChiCTR2200067033: The outcome comparison of immune tolerance induction therapy using SCT800 combined with Daratumumab or SCT800 alone in hemophilia A adolescents and adults with high titer inhibitor: a randomized controlled trial |
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| Not yet recruiting | 4 | 50 | | SCT800 combined with Daratumumab ;SCT800 alone | Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College; Hematology Hospital of Chinese Academy of Medical Sciences, Sinocelltech Ltd | hemophilia A | | | | |
ChiCTR1900027147: Efficacy and safety of adalimumab in Behcet's disease-related uveitis |
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| Not yet recruiting | 4 | 30 | | Adalimumab | State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-Sen University; State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-Sen University, None | Behcet's disease-related uveitis | | | | |
ChiCTR2200056603: The outcome comparison of low- and medium-dose immune tolerance induction therapy using domestic recombinant coagulation factor VIII in severe hemophilia A children with high titer inhibitor: a randomized controlled clinical trial |
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| Not yet recruiting | 4 | 100 | | low-dose ITI therapy(FVIII 50IU/kg QoD ;medium-dose ITI therapy(FVIII 100IU/kg QD) | Beijing Children's Hospital Affiliated to Capital Medical University; Beijing Children's Hospital Affiliated to Capital Medical University, SinoCelltech Ltd. Beijing, China | hemophilia A | | | | |
NCT03947567: Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients. |
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| Recruiting | 4 | 240 | RoW | Recombinant Human Coagulation FVIII, SCT800 | Sinocelltech Ltd. | Hemophilia A | 04/25 | 04/25 | | |
| Not yet recruiting | 4 | 270 | | Interferon Alpha-2a ;Adalimumab ;Cyclosporine | The First Affiliated Hospital of Chongqing Medical University; The First Affiliated Hospital of Chongqing Medical University, Raise independently | Uveitis in Behcet's Disease | | | | |
NCT04690322: POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies |
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| Recruiting | 4 | 28 | US | Eloctate, ELOCTATE- antihemophilic factor (recombinant), fc fusion protein, Adynovate, ADYNOVATE- antihemophilic factor (recombinant) pegylated, Emicizumab, HEMLIBRA- emicizumab injection, solution | University of Texas Southwestern Medical Center | Hemophilia A, Factor VIII | 07/25 | 07/25 | | |
NCT05744063: A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients |
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| Active, not recruiting | 4 | 13 | RoW | Emapalumab-Lzsg 5 MG/ML [Gamifant], Gamifant | Swedish Orphan Biovitrum | Primary Hemophagocytic Lymphohistiocytosis | 07/25 | 07/25 | | |
| Active, not recruiting | 4 | 46 | Europe, US, RoW | Nusinersen, ISIS 396443, BIIB058, Spinraza | Biogen | Muscular Atrophy, Spinal | 10/25 | 10/25 | | |
ChiCTR2300068826: Establishment of coagulation laboratory monitoring system in hemophilia patients treated with bispecific monoclonal antibody and clinical study |
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| Not yet recruiting | 4 | 57 | | Use drug concentration to guide treatment | Beijing Children’s Hospital, Capital Medical University; Beijing Children’s Hospital, Capital Medical University, none | hemophilia A | | | | |
NCT05936580: Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery |
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| Not yet recruiting | 4 | 28 | Europe, RoW | Nuwiq | Octapharma | Hemophilia A | 12/25 | 12/25 | | |
NuPOWER, NCT05935358: Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study |
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| Recruiting | 4 | 28 | Europe, RoW | Nuwiq | Octapharma | Severe Hemophilia A | 12/25 | 12/25 | | |
RISE, NCT05522361: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen |
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| Active, not recruiting | 4 | 10 | US | Risdiplam | Clinic for Special Children, Genentech, Inc. | Spinal Muscular Atrophy | 12/25 | 06/26 | | |
JOIN-us, NCT05856266: An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa |
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| Terminated | 4 | 9 | Europe, RoW | Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS) | Swedish Orphan Biovitrum, IQVIA Pvt. Ltd | Hemophilia A, Hemophilia B | 12/23 | 12/23 | | |
ChiCTR2300072288: Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy (SMA): a Real-World, Multi-Center Cohort Study |
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| Recruiting | 4 | 60 | | NA | Peking University First Hospital; Peking University First Hospital, Shanghai Roche Pharmaceuticals Co., Ltd. | Spinal Muscular Atrophy (SMA) | | | | |
NCT05550194: VZV in the Enteric Nervous System: Pathogenesis and Consequences |
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| Recruiting | 4 | 40 | US | ValACYclovir 1000 MG, Valtrex, Shingrix, Zoster vaccine recombinant | Vanderbilt University Medical Center, Columbia University | Achalasia | 08/26 | 08/26 | | |
ChiCTR2300073632: A clinical study on sports bleeding risk assessment model to guide Chinese children with hemophilia to improve their sports ability |
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| Not yet recruiting | 4 | 616 | | Model guidance ;no ;no ;no | Beijing Children’s Hospital, Capital Medical University; Beijing Children’s Hospital, Capital Medical University, none | hemophilia A | | | | |
NCT05888870: ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor |
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| Recruiting | 4 | 50 | RoW | SCT800 and Daratumumab, Immune tolerance induction combine anti-CD38, SCT800, Immune tolerance induction only | Institute of Hematology & Blood Diseases Hospital, China, Sinocelltech Ltd. | Hemophilia A With Inhibitor | 12/26 | 12/26 | | |