| Rare Diseases |
MOD-5017 : A phase 2a study of (Factor VIIa-CTP) in patients with haemophilia A or B |
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| Not yet recruiting | 2a | 0 | RestOfWorld | Antifibrinolytic drugs and haemostatics | OPKO | Haemophilia A | | | | |
NCT05387733: A Study to Evaluate the Efficacy and Safety of CBL-514 in Participants With Dercum's Disease Lipomas |
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| Completed | 2a | 12 | US | 10 mg CBL-514 per injection, 15 mg CBL-514 per injection | Caliway Biopharmaceuticals Co., Ltd. | Dercum's Disease | 04/23 | 04/23 | | |
| Not yet recruiting | 2 | 20 | | | St Vincent's Hospital, Sydney , St Vincent's Hospital,Sydney | Neuromyelitis Optica, Stiff Person Syndrome, Myasthenia Gravis, Central Nervous System Vasculitis | | | | |
2021-006224-40: Evaluation of thesafety and efficacy of ambroxol in Gaucher disease caused by carrying the c.1448T>C mutation (p.Leu483Pro) and other rare variants of the GBA gene, based on clinical evaluation and results of genetic and metabolomic analyses Ocena bezpieczeństwa i skuteczności stosowania ambroksolu w chorobie Gauchera spowodowanej nosicielstwem mutacji c.1448T>C (p.Leu483Pro) oraz innych rzadkich wariantów genu GBA, na podstawie oceny klinicznej i wyników analiz genetycznych i metabolomicznych |
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| Not yet recruiting | 2 | 40 | Europe | Capsule, DEFLEGMIN 75mg, kapsułki o przedłużonym uwalnianiu | Instytut ,,Pomnik-Centrum Zdrowia Dziecka", Agencja Badań Medycznych | Gaucher disease choroba Gauchera, a genetically determined disease that belongs to the group of lysosomal storage diseases, specifically sphingolipidosis choroba uwarunkowana genetycznie, należąca do grupy lizosomalnych chorób spichrzeniowych, dokładnie sfingolipidoza, Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
2021-006702-78: Open-label study to assess safety and efficacy of rapamycin in drug-resistant epilepsy associated with mTOR pathway pathologies Ocena bezpieczeństwa i skuteczności rapamycyny w leczeniu rzadkich padaczek wieku dziecięcego |
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| Not yet recruiting | 2 | 50 | Europe | Rapamune 1mg/ml oral solution, Oral solution, Rapamune 1 mg/ml oral solution | The Children's Memorial Health Institute, Medical Research Agency | epilepsy, mTORopathies, focal cortical dysplasia, LEATS padaczka, „mTORopatie”, ogniskowa dysplazja korowa, LEATS, epilepsy, mTORopathies, focal cortical dysplasia, LEATS padaczka, „mTORopatie”, ogniskowa dysplazja korowa, LEATS, Diseases [C] - Nervous System Diseases [C10] | | | | |
| Not yet recruiting | 2 | 52 | Europe | Remicade 100 mg, cyclophosphamide, Powder for solution for infusion, Remicade 100 mg, cyclophosphamide | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP), DGOS | Induction Therapy with Anti-TNF vs Cyclophosphamide in severe Behçet disease Induction Therapy with Anti-TNF vs Cyclophosphamide in severe Behçet disease, Behcet disease maladie de Behcet, Diseases [C] - Immune System Diseases [C20] | | | | |
NN Hemophilia-2: Phase II clinical trial conducting for recombinant factor VIII liposomal |
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| Ongoing | 2 | 0 | RoW | recombinant factor VIII liposomal (factor VIII liposomal) | Opperbas Holding | Hemophilia | | | | |
2005-005103-41: Clot formation and clot stability in severe haemophilia A - effect of recombinant factor VIII and tranexamic acid. |
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| Ongoing | 2 | 8 | Europe | Tranexamic acid, Recombinant factor VIII, Intravenous infusion, Cyklokapron, ReFacto, KOGENATE Bayer, Helixate NexGen, Recombinate | Anne Mette Hvas | Severe haemophilia A | | | | |
2011-000130-11: Clinical trials in rare diseases: therapeutic alternative to the use of oral corticosteroids in patients with cystic fibrosis underwent double lung transplantation. Sperimentazione clinica in malattie rare: alternativa terapeutica all'uso di corticosteroidi orali nei pazienti con Fibrosi Cistica sottoposti a Trapianto di doppio polmone. |
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| Ongoing | 2 | 20 | Europe | Dexamethasone sodium phosphate 250 mg/10 ml solution, NA, Solution for injection | AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA | Double lung transplantation in patients with Cystic Fibrosis Trapianto di doppio polmone in pazienti affetti da Fibrosi Cistica, Diseases [C] - Respiratory Tract Diseases [C08] | | | | |
ChiCTR-ONC-11001420: Study of effect of Umbilical cord Mesenchymal Stem Cell of Children with spinal muscular atrophy |
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| Completed | 2 | 10 | | stem cell transplantataion | General Hospital of Chinese people's Armed Police Forces; General Hospital of Chinese people's Armed Police Forces, General Hospital of Chinese people's Armed Police Forces | spinal muscular atrophy | | | | |
ACTRN12613001321730: Sirolimus plus prednisone for the treatment of Erdheim-Chester Disease: a pilot study |
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| Active, not recruiting | 2 | 10 | | | University of Parma, Department of Clinical and Experiment Medicine, Department of Clinical and Experimental Medicine, University of Parma | Erdheim-Chester disease | | | | |
2013-000909-24: Study of Pharmacokinetic-guided dosing of clottiing factor in Hemophilia. Studie naar farmacokinetisch gestuurde toediening van stollingsfactoren in hemofilie |
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| Ongoing | 2 | 72 | Europe | Powder and solvent for solution for injection, Powder and solution for solution for injection, , Advate, Kogenate, Helixate NexGen, Refacto, Benefix, Aafact, NovoEight | Erasmus MC, ZON-MW | Hemophillia hemofilie, hemophillia hemofilie, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
| Ongoing | 2 | 20 | Europe | hydrocortisone sodium phosphate, hydrocortisone, Solution for injection/infusion, Capsule, hydrocortisone sodium phosphate, hydrocortisone | University Hospitals Bristol NHS Foundation Trust, Medical Research Council | Addison's disease and Congenital Adrenal Hyperplasia, diseases where the body is unable to produce the hormone cortisol, Diseases [C] - Hormonal diseases [C19] | | | | |
NCT02281734: Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease and BRAF Mutation |
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| Recruiting | 2 | 18 | US | dabrafenib, BRAF inhibitor GSK2118436, GSK-2118436A, GSK2118436, Tafinlar, trametinib, GSK1120212, JTP-74057, Mekinist, laboratory biomarker analysis, quality-of-life assessment, quality of life assessment, questionnaire administration | National Cancer Institute (NCI) | Erdheim-Chester Disease | 02/15 | | | |
2011-004369-34: SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA en Pyridostigmine in volwassenen en kinderen; experimentele trial naar effect van pyridostigmine vergeleken placebo in volwassen Nederlandse patienten met spinale spieratrofie type 2, 3 en 4. |
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| Ongoing | 2 | 45 | Europe | Mestinon, RVG 03820, Tablet, pyridostigmine bromide | Universtiy Medical Center Utrecht, Prinses Beatrix Fonds | Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. Spinale spieratrofie (SMA) is een aandoening gekenmerkt door proximale spierzwakte door degeneratie van motorneuronen in de voorhoorncellen van het ruggenmerg. SMA wordt veroorzaakt door een homzygote deletie van het SMN1-gen. Er zijn 4 types SMA, geclassificeerd op basis van behaalde motorische mijlpalen en leeftijd van eerste symtpomen. De behandeling van SMA is ondersteunend., Spinal muscular atrophy (SMA) is a disorder characterized by muscle weakness caused by loss of motor cells of the spinal cord. SMA is a genentic disease. Treatment of SMA is supportive. Spinale spieratrofie (SMA) is een aandoening met proximale spierzwakte door verlies van motorische cellen in het ruggenmerg. SMA is een genetische aandoening. De behandeling van SMA is ondersteunend., Diseases [C] - Nervous System Diseases [C10] | | | | |
| Completed | 2 | 63 | RoW | Dendritic Cells DCVAC/PCa, Leuprolide acetate, Lupron, Goserelin Acetate, Zoladex | SOTIO a.s., Ascend Biopharmaceuticals Ltd | Basal Cell Carcinoma, Basal Cell Nevus Syndrome (BCNS) | 12/15 | 06/16 | | |
2005-006186-14: Open Label Phase II Evaluation of Pharmacokinetics, Efficacy, and Safety of Kedrion Human Plasma-derived Antihaemophilic Double Virus inactivated and Nanofiltered Factor IX Administered to Previously Treated Severe or Moderately Severe Hemophilia B Patients |
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| Ongoing | 2 | 10 | Europe | Nanofiltered Factor IX, | KEDRION | Severe or Moderately Severe Hemophilia B Patients | | | | |
| Ongoing | 2 | 30 | Europe | Mabthera Infusion, Solu-Medrone, Synacthen Depot, Rituximab, Methylprednisolone, Tetracosactide acetate, Mabthera infusion, Solu-Medrone, Synacthen Depot, Mabthera infusion, Solu-Medrone, Synacthen Depot | Newcastle upon Tyne Hospitals NHS Foundation Trust, Medical Research Council | Autoimmune Addison\'s disease: autoimmune primary adrenal insufficiency | | | | |
2013-004189-33: A trial comparing continuous subcutaneous hydrocortisone therapy with pulsatile subcutaneous hydrocortisone therapy in patients with Addison`s disease |
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| Ongoing | 2 | 10 | Europe | Solu-Cortef, Cortef 5 mg, Solu-Cortef, Cortef 5 mg | Haukeland University Hospital, Haukeland University Hospital, University of Bergen | Addison, s disease | | | | |
2014-002128-28: Phase 2/3 Clinical Trial with Sodium Thiosulfate for the Treatment of Calciphylaxis Phase 2/3-Studie mit Natriumthiosulfat zur Behandlung von Calciphylaxie |
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| Ongoing | 2 | 10 | Europe | Sodium Thiosulfate, STS, Natriumthiosulfat 25%, Natriumthiosulfat 25% | Dr. F. Köhler Chemie GmbH, Dr. F. Köhler Chemie GmbH | Calciphylaxis Calciphylaxie | | | | |
NCT02258243: Photodynamic Therapy Using Blue Light or Red Light in Treating Basal Cell Carcinoma in Patients With Basal Cell Nevus Syndrome |
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| Active, not recruiting | 2 | 3 | US | aminolevulinic acid hydrochloride, 5-ALA HCl, ALA HCl, aminolevulinic acid HCl, photodynamic therapy, Light Infusion Therapy™, PDT, therapy, photodynamic, laboratory biomarker analysis, questionnaire administration | Case Comprehensive Cancer Center, National Cancer Institute (NCI) | Basal Cell Carcinoma of the Skin, Nevoid Basal Cell Carcinoma Syndrome | 10/16 | 12/17 | | |
2016-003300-31: A study for the assessment of therapeutic efficacy of a drug (Denosumab) that will be administered in adult patients with mild symptoms of LCH. |
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| Ongoing | 2 | 12 | Europe | denosumab, Injection, XGEVA | Hellenic Society for the Study of Bone Metabolism, Hellenic Society for the Study of Bone Metabolis, Amgen Europe B.V. | Langerhans Cell Histiocytosis (LCH) is a rare disease of unknown etiology with variable clinical course exhibiting both neoplastic and inflammatory features. It is characterized by the accumulation and/or proliferation of specific dendritic cells resembling normal epidermal Langerhans cells., Adult patients with mild symptoms of Langerhans Cell Hystiocytosis, Diseases [C] - Immune System Diseases [C20] | | | | |
2017-003916-37: Stopping Eculizumab Treatment Safely in atypical Haemolytic Uraemic Syndrome (SETS aHUS) |
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| Not yet recruiting | 2 | 50 | Europe | Solaris, Infusion, Solaris | Newcastle Upon Tyne Hospitals NHS Foundation Trust, National Institute for Health Research | Atypical Haemolytic Uraemic Syndrome (aHUS), aHUS is a rare disease caused by a fault in the complement system. The complement system is part of your body’s immune response that attacks bugs., Diseases [C] - Immune System Diseases [C20] | | | | |
| Completed | 2 | 3 | US | AAV5-hFIXco-Padua (AMT-061) | CSL Behring | Hemophilia B | 10/18 | 09/23 | | |
2018-000160-28: Clinical study evaluating the long term safety of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 Studio clinico che valuta la sicurezza alungo termine del farmaco amifampridina fosfato in pazienti deambulanti con Atrofia Muscolare Spinale (SMA) tipo 3 |
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| Not yet recruiting | 2 | 12 | Europe | Amifampridina fosfato, [Amifampridina fosfato], Tablet, FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE | CATALYST PHARMACEUTICALS INC., Catalyst Pharmaceuticals, Inc. | Spinal Muscular Atrophy (SMA) Type 3 Atrofia Muscolare Spinale (SMA) tipo 3, Spinal Muscular Atrophy (SMA) Atrofia Muscolare Spinale (SMA), Diseases [C] - Nervous System Diseases [C10] | | | | |
BITE, NCT03010436: Benralizumab in the Treatment of Eosinophilic Granulomatosis With Polyangiitis (EGPA) Study |
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| Active, not recruiting | 2 | 10 | US | Benralizumab | National Jewish Health, AstraZeneca | Asthma | 07/19 | 12/21 | | |
2018-003903-21: A Study to Assess the pharmacokinetics and pharmacodynamics of INS1007 Administered Once Daily in Patients with Granulomatosis with Polyangiitis (GPA) |
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| Not yet recruiting | 2 | 16 | Europe | INS1007, Film-coated tablet | Insmed Incorporated, Insmed Incorporated | Granulomatosis with Polyangiitis (GPA, Wegener’s granulomatosis, Diseases [C] - Immune System Diseases [C20] | | | | |
SUNFISH, NCT02908685 / 2016-000750-35: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants |
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| Completed | 2 | 231 | Europe, Canada, Japan, US, RoW | Placebo, Risdiplam, RO7034067 | Hoffmann-La Roche | Muscular Atrophy, Spinal | 09/19 | 10/23 | | |
| Completed | 2 | 62 | Europe, Japan, US, RoW | Risdiplam, RO7034067, Evrysdi | Hoffmann-La Roche | Muscular Atrophy, Spinal | 11/19 | 12/23 | | |
2020-000971-18: A double-blind study for patients with idiopathic membranous nephropathy and severe proteinuria in treatment with ACE inhibor or angiotensin II receptor blockers. The trial will take place in hospitals in Europe. To better assess the mechanism of action of AP1189, the compound is compared to an inactive substance (placebo). The purpose of the trial is to investigate the safety and effects of the new drug compared to placebo after 12 weeks of treatment Et dobbeltblindet forsøg til patienter med idiopatisk membranøs nefropati og svær proteinuri i ACE inhibitor- eller angiotensin II-receptorblokkerende behandling. Forsøget foregår på hospitaler i Europa. For bedre at kunne vurdere AP1189s virkningsmekanisme, sammenlignes AP1189 med inaktivt stof (placebo). Formålet med forsøget er at undersøge det nye lægemiddels sikkerhed og virkninger sammenlignet med placebo efter 12 ugers behandling |
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| Not yet recruiting | 2 | 24 | Europe | AP1189, Powder for oral suspension, Tablet | SynAct Pharma ApS, SynAct Pharma ApS | Idiopathic membranous nephropathy and severe proteinuria Idiopatisk membranøs nefropati og svær proteinuri, Membranous nephropathy and severe proteinuria is a rare disease that attacks the small filters (glomeruli) in the kidney. This results in "leaks" and the kidneys lose protein in the urine. Membranøs nefropati er en sjælden sygdom, som angriber de små filtre (glomeruli) i nyrerne. Dette resulterer i "utætheder", hvor nyrerne taber protein i urinen., Diseases [C] - Immune System Diseases [C20] | | | | |
2019-003830-17: Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 Inhibitor |
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| Not yet recruiting | 2 | 26 | Europe | ALXN2050, ACH-0145228, Capsule | Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Achillion Pharmaceuticals, Inc. a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Nocturnal Hemoglobinuria (PNH), Diseases [C] - Immune System Diseases [C20] | | | | |
2020-003268-25: Hydroxychloroquine in isolated cutaneous mastocytosis patients or indolent systemic mastocytosis with associated skin involvement patients: proof of concept study Hydroxychloroquine dans la mastocytose cutanée isolée ou systémique indolente avec atteinte cutanée associée : étude de preuve de concept |
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| Not yet recruiting | 2 | 30 | Europe | Plaquenil (Hydroxychloroquineà, Tablet, Plaquenil | CHU Toulouse, GIRGI-SOHO | mastocytosis mastocytose, mastocytosis mastocytose, Diseases [C] - Skin and Connective Tissue Diseases [C17] | | | | |
2020-002483-31: Phase II study to evaluate 68GaNOTA-Anti-MMR-VHH2 , a new tracer for positron emission tomography (PET/CT) of macrophages in oncological, hematological,cardiovascular diseases and sarcoidosis. |
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| Not yet recruiting | 2 | 140 | Europe | 68Ga-NOTA-Anti-MMR-VHH2, Solution for injection | UZ Brussel, UZ Brussel | C1: malignancies of the head and neck. C2: malignancy with a solid componentC3: carotid plaque, planned for (SOC) carotid endarterectomy.C4: biopsy-proven Hodgkin or non-Hodgkin lymphoma.C5: suspected for hemophagocytic lymphohistiocytosis (HLH), planned for (SOC) bone marrow biopsy in case it is not done before.C6: biopsy proven or suspected endomyocardial sarcoidosis C7: biopsy proven sarcoidosis, Head-and-Neck cancer, cancer with solid lesions, atherosclerosis of large artery in the neck, Hodgkin/non-Hogdkin Lymphoma, hemophagocytic lymphohistiocytosis, cardiac sarcoidosis and sarcoidosis., Diseases [C] - Cancer [C04] | | | | |
| Not yet recruiting | 2 | 55 | NA | SRK-015 | Scholar Rock, Inc., Scholar Rock, Inc. | Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations | 01/21 | 04/21 | | |
| Completed | 2 | 58 | Europe, US | SRK-015 | Scholar Rock, Inc. | Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations | 01/21 | 02/24 | | |
2020-005005-17: Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy |
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| Not yet recruiting | 2 | 24 | Europe | Pozelimab, Cemdisiran, REGN3918, ALN-CC5, Solution for injection/infusion, Solution for injection | Regeneron Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria, Paroxysmal Nocturnal Hemoglobinuria (PNH), Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
NCT01573754: Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda |
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| Completed | 2 | 48 | US | Hydroxychloroquine, Plaquenil, Phlebotomy | The University of Texas Medical Branch, Galveston | Porphyria Cutanea Tarda | 07/21 | 07/21 | | |
2020-003807-32: A study to find out if BT200 is safe, well-tolerated and has effects in patients with hereditary bleeding disorders |
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| Not yet recruiting | 2 | 25 | Europe | BT200, Solution for injection | Medical University of Vienna, Band Therapeutics LLC | - Patients with severe congenital hemophilia A- Patients with mild-moderate hemophilia A- Heterozygous carriers of hemophilia A- Patients with VWD Type 1, e.g. “Vicenza” type- Acquired Von Willebrand Syndrome (aVWS) without specific inhibitor- Patients with VWD Type 2b- Patients with VWD Type 3 on substitution VWF therapy, Patients with hereditary bleeding disorders, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2021-000679-35: SATELITE “Salvage Therapy for Patients with Inadequate Response to Standard of Care Therapy in Granulomatosis with Polyangiitis” SATELITE "Traitement de sauvetage pour les patients ayant une réponse inadéquate au traitement de référence dans la granulomatose avec polyangéite" |
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| Not yet recruiting | 2 | 42 | Europe | Concentrate for solution for infusion, Solution for injection, Tablet, Rituximab, ROACTEMRA, ORENCIA, Methotrexate, Mycophénolate mofétil, Azathioprine | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP), ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS | Granulomatosis with Polyangiitis Granulomatose avec polyangéite, Granulomatosis with Polyangiitis Granulomatose avec polyangéite, Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
2021-000407-20: Treatment of nonsevere sporadic Hemophagocytosis Lymphohistiocytosis (HLHs) with ITACITINIB: a phase II prospective trial. |
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| Not yet recruiting | 2 | 63 | Europe | Itacitinib, Tablet | ASSISTANCE PUBLIQUE HOPITAUX DE PARIS, INCYTE BIOSCIENCES INTERNATIONAL SARL Company, | Adults patients having non severe sporadic Hemophagocytosis Lymphohistiocytosis, Adults patients having non severe sporadic Hemophagocytosis Lymphohistiocytosis, Diseases [C] - Immune System Diseases [C20] | | | | |
ChiCTR2000029977: Study for Ruxolitinib as a front-line Therapy of pediatric Hemophagocytic Lymphohistiocytosis |
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| Recruiting | 2 | 12 | | 5 mg or 10 mg twice daily depending on the body weight (<=20kg or＞20kg, respectively) by oral for 28 days | Beijing Children's Hospital, Capital Medical University, National Center for Children's Health; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, self-raised funds | hemophagocytic lymphohistiocytosis | | | | |
ChiCTR2000039675: Phase II clinical trial of the superiority of all-trans retinoic acid in the treament of paroxysmal nocturnal hemoglobinuria |
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| Recruiting | 2 | 20 | | Tretinoin 0.33mg/kg.d; methylprednisolone 0.8mg/kg.d ;methylprednisolone 0.8mg/kg.d | Department of Hematology, Second Medical Center, Chinese PLA General Hospital; Chinese PLA General Hospital, Hospital research projects | paroxysmal nocturnal hemoglobinuria | | | | |
NCT03982771: BCD Regimen in Newly Diagnosed Idiopathic Multicentric Castleman's Disease (iMCD) |
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| Recruiting | 2 | 30 | RoW | Bortezomib, Cyclophosphamide, Dexamethason | Peking Union Medical College Hospital | Idiopathic Multicentric Castleman's Disease | 01/22 | 01/23 | | |
NCT04592692: A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip |
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| Recruiting | 2 | 20 | RoW | PEGylated Liposome (PEGLip), Simoctocog alfa | Ascension Healthcare Development Limited | Hemophilia A With Inhibitor | 02/22 | 05/22 | | |
| Active, not recruiting | 2 | 10 | Europe | OTL-200, Previously GSK2696274 | Orchard Therapeutics, Ospedale San Raffaele | Lysosomal Storage Disease, Metachromatic Leukodystrophy | 04/22 | 04/28 | | |
NCT04614454: High Frequency Impulse Therapy for Neuropathic Pain in NMOSD |
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| Active, not recruiting | 2 | 46 | US | High Frequency Impulse Therapy, Transcutaneous electrical nerve stimulation, High Frequency Impulse Therapy - Sham, Transcutaneous electrical nerve stimulation - Sham | Massachusetts General Hospital | Neuromyelitis Optica | 04/22 | 12/24 | | |
NCT04888507: Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy |
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| Completed | 2 | 6 | Europe | Pozelimab, REGN3918, Cemdisiran, ALN-CC5 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 05/22 | 05/23 | | |
| Active, not recruiting | 2 | 20 | US | Abatacept, Orencia, Placebo | Rohit Aggarwal, MD, Bristol-Myers Squibb | Myositis, Interstitial Lung Disease | 05/22 | 07/23 | | |
ChiCTR2000041490: A single-arm, open-label, phase 2 clinical trial of Tislelizumab combined with Lucitanib for secondary hemophagocytic lymphohistiocytosis. |
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| Recruiting | 2 | 29 | China | Baizean (tislelizumab) - BeiGene, Novartis, lucitanib (E 3810) - Clovis, Servier | Institute of Hematology & Hospital of Blood DiseasesChinese Academy of Medical Sciences & Peking Union Medical College | Hemophagocytic lymphohistiocytosis;Oncoogy | | | | |
NCT04670770: An Open Label Study of the Effects of SHR1459 in NMOSDs Patients |
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| Completed | 2 | 10 | RoW | Drug - SHR1459 | Reistone Biopharma Company Limited | Neuromyelitis Optica Spectrum Disorders | 08/22 | 08/22 | | |
NCT05265286: A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection |
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| Completed | 2 | 15 | RoW | FRSW117, PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection | Jiangsu Gensciences lnc. | Severe Hemophilia A | 08/22 | 08/22 | | |
NCT03333486: Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer |
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| Active, not recruiting | 2 | 31 | US | Cyclophosphamide, (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719, Fludarabine Phosphate, 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara, SH T 586, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, peripheral stem cell support, Peripheral Stem Cell Transplant, peripheral stem cell transplantation, Total-Body Irradiation, TOTAL BODY IRRADIATION, Whole-Body Irradiation | Roswell Park Cancer Institute | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome | 08/23 | 08/24 | | |
NCT05098067: Capsaicin Cream as an Adjunctive Therapy for Nausea and Vomiting of Pregnancy |
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| Completed | 2 | 30 | US | Capsaicin Topical Cream, Metoclopramide, Reglan, Ondansetron, Zofran, Lactated Ringers, Intravenous | Women and Infants Hospital of Rhode Island | Hyperemesis Gravidarum, Nausea Gravidarum, Vomiting of Pregnancy | 10/22 | 03/23 | | |
NCT04811716: Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy |
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| Completed | 2 | 24 | Europe, RoW | Pozelimab, REGN3918, Cemdisiran, ALN-CC5 | Regeneron Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | 10/22 | 10/23 | | |
NCT05345522: A Study of Anti-IL-6R mAb Injection in Patients With iMCD |
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| Recruiting | 2 | 9 | RoW | Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 4mg/kg, Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 6mg/kg, Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 8mg/kg | Beijing VDJBio Co., LTD. | Idiopathic Multicentric Castleman's Disease | 11/22 | 12/24 | | |
NCT04218565: Golimumab for the Treatment of Refractory Behcet's Uveitis |
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| Terminated | 2 | 1 | RoW | Golimumab (GOL) | Wenjie Zheng | Behcet Syndrome, Uveitis | 12/22 | 12/22 | | |
ChiCTR2000031702: The role of ruxolitinib in the treatment of hemophagocytic lymphohistiocytosis (HLH) in children |
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| Recruiting | 2 | 200 | | Patients received oral ruxolitinib dosed on 2.5mg, 5 mg or 10 mg twice daily depending on the body weight (≤10kg,≤20kg or＞20kg, respectively) | Beijing Children's Hospital Affiliated to Capital Medical University, National Center for Children's Health; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, self-raised funds | Hemophagocytic lymphohistiocytosis | | | | |
ChiCTR2200058864: A single-arm, open, multicenter Phase IIa clinical study to evaluate the efficacy and safety of recombinant humanized anti-interleukin-6 receptor monoclonal antibody injection in patients with idiopathic multicenter Castleman's disease (iMCD) |
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| Not yet recruiting | 2 | 9 | | Injection VDJ006 | Peking Union Medical College Hospital, Chinese Academy of Medical Sciences; Beijing Weidjie Biotechnology Co., LTD, Self-raising | IMCD | | | | |
2019-004430-42: Evaluation of the effect of emicizumab in patients suffering from acute bleeds with previously no family history. |
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| Not yet recruiting | 2 | 47 | Europe | Solution for injection, Hemlibra® | GWT-TUD GmbH, F. Hoffmann-La Roche Ltd | Acquired Hemophilia A (AHA), aquired bleeding disorder, Body processes [G] - Immune system processes [G12] | | | | |
2018-004406-25: To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). |
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| Not yet recruiting | 2 | 39 | Europe, RoW | N-Acetyl-L-Leucine, IB1001, Powder for oral suspension, Granules for oral suspension | IntraBio Ltd, IntraBio Ltd, | To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)., Tay–Sachs disease (α-mutations) and Sandhoff disease (β-mutations) are neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD)., Diseases [C] - Nervous System Diseases [C10] | | | | |
| Ongoing | 2 | 200 | Europe | BCX9930, BCX9930, Tablet | BioCryst Pharmaceuticals Inc, BioCryst Pharmaceuticals Inc | Paroxysmal Nocturnal Hemoglobinuria, PNH, Diseases [C] - Immune System Diseases [C20] | | | | |
| Active, not recruiting | 2 | 26 | Europe, US, RoW | Risdiplam, Evrysdi | Hoffmann-La Roche | Muscular Atrophy, Spinal | 02/23 | 03/27 | | |
2022-002395-36: A study of open administration of a drug, which removes iron from the body of patients with transfusion-dependent β-thalassemia, aiming to find the most appropriate dose through use of different dose levels in different patients |
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| Ongoing | 2 | 90 | Europe | SP-420, SP-420, Capsule, hard | Pharmacosmos A/S, Pharmacosmos A/S | Transfusion-dependent β-thalassemia, Beta-thalassemia is a rare disease that is inherited and leads to a reduced production of hemoglobin (anemia)., Diseases [C] - Blood and lymphatic diseases [C15] | | | | |
| Not yet recruiting | 2 | 98 | Europe | Etidronate, Etidronate, Capsule | University Medical Center Utrecht, Hersenstichting | Fahr’s disease or sondrome is a neurodegenerative disease in which all patients present with bilateral vessel associated calcifications in the basal ganglia in the absence of other secondary causes of brain calcifications. The clinical penetration of Fahr's disease or syndrome is incomplete and heterogeneous comprising of neuropsychiatric signs, cognitive decline, and movement disorders. The symptoms start between 30 and 50 years and are (slowly) progressive., Patients with Fahr's disease or syndrome have calcifications in specific areas in the brain. This results in mobility problems, disturbed mental functions and/or psychiatric problems., Diseases [C] - Cardiovascular Diseases [C14] | | | | |
| Active, not recruiting | 2 | 31 | Europe | Belimumab, Benlysta, Rituximab, Truxima, Prednisolone, prednisone | Rachel Jones, GlaxoSmithKline, Medical Research Council, Imperial College London, University College, London, Newcastle University, University of Glasgow, University of Cambridge | ANCA Associated Vasculitis, Granulomatosis With Polyangiitis | 04/23 | 11/23 | | |
2021-000854-24: Phase II multicenter clinical trial, with treatment randomly assigned, in which patient nor physician are aware of the assigned treatment, active or placebo, to evaluate efficacy and safety of Velusetrag 15 mg (3 x 5 mg capsule) in patients with Chronic Intestinal Pseudo-Obstruction (CIPO) Ensayo clínico multicéntrico de fase II, con tratamiento asignado aleatoriamente, en el que el paciente ni el médico conocen el tratamiento asignado, activo o placebo, para evaluar la eficacia y seguridad de Velusetrag 15 mg (3 cápsulas de 5 mg) en pacientes con pseudoobstrucción intestinal crónica (CIPO). |
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| Not yet recruiting | 2 | 16 | Europe | Velusetrag, [TD-5108 hydrochloride], Capsule, hard | ALFASIGMA S.P.A., Alfasigma S.p.A. | Chronic intestinal pseudo-obstruction (CIPO) is a rare, severe condition characterized by an impairment of coordinated propulsive activity in the intestinal tract resulting in a clinical picture similar to that of mechanical intestinal obstruction, although in the absence of any lesion occluding the gut. CIPO can be idiopathic, when no primary underlying disorder is demonstrated, or secondary, when related to systemic diseases. La pseudoobstrucción intestinal crónica (CIPO) es una enfermedad rara y grave, caracterizada por una alteración de la actividad de propulsión coordinada en los intestinos que se traduce en un cuadro clínico parecido al de la obstrucción intestinal mecánica, salvo por la ausencia de lesiones que ocluyen el intestino. La CIPO puede ser idiopática, cuando no hay un trastorno primario subyacente demostrado, o secundaria, cuando está relacionada con enfermedades sistémicas., Chronic intestinal pseudo-obstruction (CIPO) is a rare disease impacting the intestinal motility, potentially invalidating. La pseudoobstrucción intestinal crónica (CIPO) es una enfermedad rara que afecta a la motilidad intestinal, potencialmente invalidante., Diseases [C] - Digestive System Diseases [C06] | | | | |
2023-000128-12: Single-center phase II study on the use of electrochemotherapy in the treatment of Paget's disease and high-grade or initially invasive precancerous squamous lesions of the vulva (GinOnc-ECT study) Studio di fase II monocentrico sull’utilizzo dell’elettrochemioterapia nel trattamento del M. di Paget e delle lesioni squamose precancerose di alto grado o inizialmente invasive della vulva (GinOnc-ECT study) |
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| Not yet recruiting | 2 | 18 | Europe | cisplatino, Bleomicina, NA, Concentrate for solution for injection/infusion, Powder for solution for injection/infusion | FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE | patients with newly diagnosed or recurrent high-grade or initially invasive precancerous squamous lesions of the vulva and non-invasive vulvar Paget's disease pazienti con nuova diagnosi o recidiva di lesioni squamose precancerose di alto grado o inizialmente invasive della vulva e di malattia di Paget vulvare non invasivo, patients with newly diagnosed or recurrent high-grade or initially invasive precancerous squamous lesions of the vulva and non-invasive vulvar Paget's disease pazienti con nuova diagnosi o recidiva di lesioni squamose precancerose di alto grado o inizialmente invasive della vulva e di malattia di Paget vulvare non invasivo, Diseases [C] - Cancer [C04] | | | | |
NCT04170023: Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy |
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| Terminated | 2 | 29 | Europe, Canada, RoW | ALXN2050, ACH-0145228 | Alexion Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 04/23 | 03/24 | | |
2022-003853-70: Tirzepatide, a new drug for type 2 diabetes mellitus and obesity, as a possible treatment to modify the clinical evolution of Wolfram syndrome type 1 Tirzepatide, un nuovo farmaco per il diabete mellito di tipo 2 e l’obesità, come possibile trattamento per modificare l’evoluzione clinica della sindrome di Wolfram di tipo 1 |
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| Not yet recruiting | 2 | 10 | Europe | MOUNJARO - tirzepatide, [MOUNJARO], Solution for injection, MOUNJARO | OSPEDALE SAN RAFFAELE, PNRR - Ministero della Salute | Wolfram syndrome type 1 Sindrome di Wolfram tipo 1, Diabetes mellitus, optic nerve atrophy, sensorineural deafness, diabetes insipidus, and neurodegeneration in variable combination with poor prognosis Diabete mellito, atrofia dei nervi ottici, sordità neurosensoriale, diabete insipido e neurodegenerazione in combinazione variabilie e a prognosi infausta, Diseases [C] - Hormonal diseases [C19] | | | | |
NCT05421429: KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia |
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| Recruiting | 2 | 24 | RoW | KN057 doseⅠ, KN057 dose Ⅱ, KN057 dose Ⅲ | Suzhou Alphamab Co., Ltd. | Hemophilia | 02/24 | 06/24 | | |
NCT04416516: Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor |
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| Completed | 2 | 21 | RoW | ASN-002 | Ascend Biopharmaceuticals Ltd | Basal Cell Carcinoma, Basal Cell Nevus Syndrome | 02/24 | 02/24 | | |
CAMPUS, NCT04064944: Comparison of the Efficacy and Safety of Immunoadsorption and Plasma Exchange for Acute Attack of Refractory Neuromyelitis Optica Spectrum Disorders |
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| Not yet recruiting | 2 | 144 | NA | blood purification equipment | Third Affiliated Hospital, Sun Yat-Sen University, Zhongshan Ophthalmic Center, Sun Yat-sen University, Guangdong 999 Brain Hospital | Neuromyelitis Optica Spectrum Disorder | 08/23 | 12/24 | | |
NCT05619926: Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor |
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| Completed | 2 | 32 | RoW | STSP-0601 for Injection | Staidson (Beijing) Biopharmaceuticals Co., Ltd, Beijing Novikang Medical Technology Co., LTD | Hemophilia | 08/23 | 08/23 | | |
| Completed | 2 | 275 | Europe, Japan, US, RoW | NNC0365-3769 (Mim8), Placebo (Mim8) | Novo Nordisk A/S | Healthy Volunteers, Haemophilia A With or Without Inhibitors | 10/23 | 10/23 | | |
RILUSCI, NCT02859792: Riluzole in the Treatment of Spasticity in the Traumatic Chronic Spinal Cord Injury Condition |
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| Recruiting | 2 | 90 | Europe | Riluzole, Placebo, Blood Samples | Assistance Publique Hopitaux De Marseille | Spinal Cord Injury | 11/23 | 12/23 | | |
| Recruiting | 2 | 63 | Europe | Itacitinib | Assistance Publique - Hôpitaux de Paris, Incyte Corporation | Adults Patients Having Non Severe HLH | 11/23 | 02/24 | | |
| Recruiting | 2 | 36 | Canada, US | Naltrexone Hydrochloride, Placebo Comparator | University of Pennsylvania | Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Giant Cell Arteritis, Granulomatosis With Polyangiitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis | 12/24 | 12/24 | | |
NCT03933904: Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease |
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| Recruiting | 2 | 24 | US | Sirolimus, Rapamune, Rapamycin | University of Pennsylvania | Castleman Disease, Castleman's Disease, Multicentric | 06/24 | 06/25 | | |
NCT04924647: Lenalidomide and Dexamethasone for Rosai-Dorfman Disease |
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| Recruiting | 2 | 16 | RoW | lenalidomide and dexamethasone | Peking Union Medical College Hospital | Rosai-Dorfman Disease | 12/23 | 12/24 | | |
NCT04846348: Topical Composition Therapy for the Treatment of Cutaneous Mastocytosis |
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| Terminated | 2 | 1 | US | Topical preparation of sodium cromolyn 5%, diphenhydramine 1% and trolamine salicylate 10% in emollient cream base., Skin Guard | Joseph Butterfield | Cutaneous Mastocytoses | 09/21 | 09/21 | | |
NCT06050226: A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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| Recruiting | 2 | 40 | RoW | MY008211A tablets, MY008211A | Wuhan Createrna Science and Technology Co., Ltd | Paroxysmal Nocturnal Hemoglobinuria | 01/24 | 07/24 | | |
| Not yet recruiting | 2 | 50 | | ICIs | the second hospital of Dalian Medical University ; the second hospital of Dalian Medical University, the second hospital of Dalian Medical University “1+X” Project | PCNSL | | | | |
NCT02523040: A Study of Lenalidomide for Adult Histiocyte Disorders |
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| Active, not recruiting | 2 | 12 | US | Lenalidomide, Revlimid | Dana-Farber Cancer Institute, Celgene | Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease, Histiocytic Sarcoma (HS) | 02/24 | 08/24 | | |
| Recruiting | 2 | 48 | Europe | Immune Globulin Intravenous (Human), Nanogam, Placebo, Sodium chloride or saline 0.9% | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA), Princess Beatrix Muscle Foundation | Inflammatory Myopathy, Idiopathic, Dermatomyositis, Antisynthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Polymyositis, Myositis | 02/24 | 09/24 | | |
NCT06160791: Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH) |
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| Not yet recruiting | 2 | 36 | US | Ruxolitinib, INCB18424, Oral JAK, Etoposide, Toposar, Lastet, Dexamethasone, Dexa, Non-interventional Imaging, Ultrasound, Magnetic resonance imaging (MRI), Research Biopsy, Biopsy, Biospecimen Collection, Specimen collection | University of California, San Francisco, Incyte Corporation | Hemophagocytic Lymphohistiocytoses | 05/27 | 05/27 | | |
| Recruiting | 2 | 50 | US | Mirdametinib | Children's Hospital Medical Center, Cincinnati | Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG), Rosai-Dorfman Disease (RDD), Histiocytic Disorders | 03/30 | 03/31 | | |
| Recruiting | 2 | 24 | RoW | HRS-5965 tablets | Chengdu Suncadia Medicine Co., Ltd. | Paroxysmal Nocturnal Hemoglobinuria | 12/27 | 12/27 | | |
NCT06298955: Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria |
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| Recruiting | 2 | 25 | Europe, RoW | OMS906 study drug | Omeros Corporation | Paroxysmal Nocturnal Hemoglobinuria | 12/26 | 04/27 | | |
| Terminated | 2 | 12 | RoW | BCX9930 monotherapy, Placebo | BioCryst Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 09/23 | 09/23 | | |
| Not yet recruiting | 2 | 60 | NA | CE-VST01-JC | Cellevolve Bio Inc | Progressive Multifocal Leukoencephalopathy | 03/24 | 04/25 | | |
| Recruiting | 2 | 60 | Europe | Filgotinib | UMC Utrecht, Alfasigma S.p.A., ReumaNederland, Autoimmune Research and Collaboration Hub | Behcet's Disease, Idiopathic Inflammatory Myopathies, IgG4-related Disease | 12/26 | 12/26 | | |
| Recruiting | 2 | 25 | RoW | STSP-0601 for Injection | Staidson (Beijing) Biopharmaceuticals Co., Ltd | Hemophilia | 02/25 | 02/25 | | |
| Recruiting | 2 | 12 | Europe, US, RoW | Pegcetacoplan | Apellis Pharmaceuticals, Inc., Apellis Pharmaceuticals, Inc. | Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria | 04/24 | 10/24 | | |
NCT05065567: Haloperidol, Droperidol, Ondansetron in Cannabis Hyperemesis |
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| Terminated | 2 | 38 | US | Droperidol, Haloperidol, Ondansetron 8mg | Spectrum Health - Lakeland | Cyclic Vomiting Syndrome | 11/23 | 11/23 | | |
| Not yet recruiting | 2 | 28 | NA | Multi-ingredient supplement (PDT-MIS), Placebo (PLA) | McMaster University | Pompe Disease, Muscle Loss, Obesity, Nutrition Poor, Lysosomal Storage Diseases, Glycogen Storage Disease Type II, Glycogen Storage Disease Type II Late Onset, Glycogen Storage Disease Type II, Adult | 09/24 | 04/25 | | |
RACEMATE, NCT06230354: Explore the Efficacy and Mechanism of Action of Tezepelumab in Eosinophilic Granulomatosis With Polyangiitis |
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| Enrolling by invitation | 2 | 66 | Europe | Tezepelumab, Placebo | Imperial College London, AstraZeneca | EGPA - Eosinophilic Granulomatosis With Polyangiitis | 10/25 | 10/25 | | |
NCT06144086: A Phase 2 Trial of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia (OP-724-H201) |
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| Recruiting | 2 | 6 | Japan | Foscenvivint, CBP-beta-catenin inhibitor, OP-724, PRI-724 (former name) | Kiminori Kimura, MD, Japan Agency for Medical Research and Development | Liver Cirrhosis | 03/25 | 06/25 | | |
CONSENTII, NCT03427060: Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms |
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| Enrolling by invitation | 2 | 6 | US | Coversin | AKARI Therapeutics | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 06/24 | 06/24 | | |
NCT05195762: 12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi |
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| Withdrawn | 2 | 12 | US | NFX-179 Gel 1.50% | Albert Chiou, NFlection Therapeutics, Inc. | Epidermal Nevi, Nevus Sebaceus | 06/24 | 12/24 | | |
NCT06412497: MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. |
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| Recruiting | 2 | 60 | US | Rituximab, Rabbit ATG, Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, TBI, Cell Infusion, Post-Transplant G-CSF, Filgrastim, Tacrolimus, Mycophenolate Mofetil, MMF | Masonic Cancer Center, University of Minnesota | Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria | 05/35 | 05/36 | | |
