What’s New in Research
Every year, hundreds of new clinical trials are initiated to test new “experimental” drugs, or to test new uses of already approved drugs on humans, in a wide range of disease areas. These trials are conducted by researchers in countries all around the world, including Canada, in locations like hospitals, universities, doctors’ offices, and community clinics. Through voluntary participation in clinical trials, patients get a chance to take part in research that could improve their health and help them access a drug, prior to its approval. Like all drugs, the ones used in clinical trials have potential benefits as well as risks, and since they are still being studied, there is usually limited information about safety and efficacy. Before deciding to take part in a clinical trial, discuss the potential risks and benefits with your health care provider, so that you can make an informed decision about your health.
There is an unprecedented number of clinical trials currently underway in PNH, including:
ClinicalTrials.gov: Paroxysmal Nocturnal Hemoglobinuria | Last update posted in the last 14 days
- Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal HemoglobinuriaCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: HRS-5965 tabletsSponsor: Chengdu Suncadia Medicine Co., Ltd.Not yet recruiting
- A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Condition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: MY008211A tabletsSponsor: Wuhan Createrna Science and Technology Co., LtdRecruiting
- Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in ChinaCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: EculizumabSponsors: Alexion; AstraZenecaRecruiting
- Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal HemoglobinuriaCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: BCX9930Sponsor: BioCryst PharmaceuticalsRecruiting
- Study of NM8074 in Adult PNH Patients With Inadequate Response to SolirisCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: NM8074; Drug: SolirisSponsor: NovelMed TherapeuticsNot yet recruiting
- Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: NM8074Sponsor: NovelMed TherapeuticsNot yet recruiting
- A C5 Inhibitor-controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor TherapyCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Ravulizumab; Drug: Pozelimab; Drug: Cemdisiran; Drug: EculizumabSponsor: Regeneron PharmaceuticalsRecruiting
- Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Conditions: Bone Marrow Failure Syndrome; Congenital Amegakaryocytic Thrombocytopenia; Congenital Pure Red Cell Aplasia; Hereditary Sideroblastic Anemia; Myeloid Neoplasms With Germline GATA2 Mutation; Paroxysmal Nocturnal Hemoglobinuria; Shwachman-Diamond SyndromeInterventions: Drug: Treosulfan; Drug: Fludarabine Phosphate; Drug: Tacrolimus; Drug: Methotrexate; Biological: Lapine T-Lymphocyte Immune Globulin; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Allogeneic Bone Marrow Transplantation; Other: Quality-of-Life AssessmentSponsors: Fred Hutchinson Cancer Center; Blood and Marrow Transplant Clinical Trials Network; National Cancer Institute (NCI); National Marrow Donor Program; National Heart, Lung, and Blood Institute (NHLBI)Recruiting
- Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®Condition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Biological: Elizaria®Sponsor: AO GENERIUMCompleted
- A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: CrovalimabSponsor: Hoffmann-La RocheActive, not recruiting