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 Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Condition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: PegcetacoplanSponsor: Swedish Orphan BiovitrumNot yet recruiting
- Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as MonotherapyCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention: Drug: ALXN2050Sponsor: AlexionActive, not recruiting
- A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal HemoglobinuriaCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Biological: RavulizumabSponsor: AlexionCompleted
- ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Interventions: Biological: Ravulizumab; Biological: EculizumabSponsor: AlexionCompleted
- 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