Clinical Trials:
Pyruvate Kinase Deficiency (PKD)
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. PKD impacts an estimated 4,000-8,000 individuals in the U.S. and Europe.
Enrollment in the global Phase 1 open-label, single-arm clinical trial (NCT04105166) is complete, with four adult and pediatric transfusion-dependent PKD patients enrolled in the U.S. and EU.
Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com.
For more details about the trial, visit clinicaltrials.gov.
To read our Expanded Access statement, click here.