Clinical Trials:
Pyruvate Kinase Deficiency
RP-L301 is currently being developed for PKD, a rare blood disorder characterized by excessive rupture of red blood cells, resulting in frequent, chronic anemia that may be severe or life-threatening for some patients.
The global Phase 1 open-label, single-arm clinical trial (NCT04105166) will enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. It will evaluate safety and efficacy of the treatment.
For more details about the trial, visit clinicaltrials.gov.
For more information about PKD, visit our Patients and Community page or email us at clinicaltrials@nullrocketpharma.com.