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
- BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor TherapyCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Interventions: Drug: BCX9930; Drug: Eculizumab; Drug: RavulizumabSponsor: BioCryst PharmaceuticalsRecruiting
- BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor TherapyCondition: Paroxysmal Nocturnal Hemoglobinuria (PNH)Interventions: Drug: BCX9930 monotherapy; Drug: PlaceboSponsor: BioCryst PharmaceuticalsRecruiting
- Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)Condition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: Danicopan; Drug: Placebo; Drug: C5 InhibitorSponsor: Alexion 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 PharmaceuticalRecruiting
- 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
- A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNHCondition: Paroxysmal Nocturnal HemoglobinuriaInterventions: Drug: ABP 959; Drug: EculizumabSponsor: AmgenActive, not recruiting
- 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 PharmaceuticalsCompleted
- 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