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 Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNHCondition: Paroxysmal Nocturnal HemoglobinuriaIntervention: Drug: DanicopanSponsor: Alexion PharmaceuticalsNot yet recruiting
- 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
- 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
- Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisConditions: Severe Aplastic Anemia (SAA); Hypo-Plastic Myelodysplastic Syndrome (MDS); Paroxysmal Nocturnal Hemoglobinuria (PNH)Interventions: Drug: Cyclophosphamide; Other: Peripheral Blood Stem CellsSponsor: National Heart, Lung, and Blood Institute (NHLBI)Recruiting
- Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)Condition: PNHIntervention: Biological: ALXN1210Sponsor: Alexion PharmaceuticalsCompleted
- 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