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
- A Study of Single-dose MY008211A in Healthy AdultsCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: MY008211A tablets; Drug: PlaceboSponsor: Wuhan Createrna Science and Technology Co., LtdActive, not recruiting
- This is a Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.Condition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: IptacopanSponsor: Novartis PharmaceuticalsNot yet recruiting
- Ravulizumab-Controlled Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor TherapyCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Ravulizumab; Drug: Pozelimab; Drug: CemdisiranSponsor: Regeneron PharmaceuticalsRecruiting
- Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention: Drug: Iptacopan (LNP023)Sponsor: Novartis PharmaceuticalsActive, not recruiting
- Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Interventions: Drug: LNP023; Drug: Eculizumab; Drug: RavulizumabSponsor: Novartis PharmaceuticalsActive, not recruiting
- A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement InhibitorsCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Crovalimab; Drug: EculizumabSponsors: Hoffmann-La Roche; Chugai PharmaceuticalRecruiting
- Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal HemoglobinuriaCondition: Paroxysmal Hemoglobinuria, NocturnalInterventions: Drug: Crovalimab; Drug: PlaceboSponsor: Hoffmann-La RocheActive, not recruiting
- Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsConditions: Severe Aplastic Anemia; MDS (Myelodysplastic Syndrome)Interventions: Device: Miltenyi CD34 Reagent System; Other: Donor derived G-CSF mobilized PBCSponsor: National Heart, Lung, and Blood Institute (NHLBI)Recruiting