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
- To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104Condition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: KP104; Drug: PlaceboSponsor: Kira Pharmacenticals (US), LLC.Active, not recruiting
- To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104Condition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: KP104Sponsor: Kira Pharmacenticals (US), LLC.Not yet recruiting
- 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 PharmaceuticalsRecruiting
- Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: IptacopanSponsor: Novartis PharmaceuticalsRecruiting
- A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.Condition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Crovalimab; Drug: EculizumabSponsors: Hoffmann-La Roche; Chugai PharmaceuticalActive, not recruiting
- A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors.Condition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Crovalimab; Drug: EculizumabSponsors: Hoffmann-La Roche; Chugai PharmaceuticalRecruiting
- Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical StudyCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention: Drug: Zilucoplan (RA101495)Sponsor: Ra PharmaceuticalsTerminated
- 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
- Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH PatientsCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention: Drug: Zilucoplan (RA101495)Sponsor: Ra PharmaceuticalsCompleted
- Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to EculizumabCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Intervention: Drug: Zilucoplan (RA101495)Sponsor: Ra PharmaceuticalsCompleted