Paroxysmal Nocturnal Hemoglobinuria
NCI Definition: A rare acquired hematologic disorder characterized by hemolytic anemia, dark-colored urine due to the release of hemoglobin in the blood, and thrombosis. The episodes of hemolysis tend to occur at night. It is caused by a somatic mutation in the glycosylphosphatidylinositol biosynthesis gene. 
There are 2 clinical trials for paroxysmal nocturnal hemoglobinuria, of which 1 is open and 1 is completed or closed. Of the trials that contain paroxysmal nocturnal hemoglobinuria as an inclusion criterion, 1 is phase 2 (0 open) and 1 is no phase specified (1 open).
Allogeneic hematopoietic stem cell transplantation, cladribine, and melphalan are the most common interventions in paroxysmal nocturnal hemoglobinuria clinical trials.
2. The AACR Project GENIE Consortium. AACR Project GENIE: powering precision medicine through an international consortium. Cancer Discovery. 2017;7(8):818-831. Dataset Version 8. This dataset does not represent the totality of the genetic landscape; see paper for more information.