Clinical Trials:
Pyruvate Kinase Deficiency (PKD)
RP-L301 is currently being developed for PKD, a rare, genetic 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.
A single arm, open-label, global Phase 2 trial to evaluate the efficacy and safety of RP-L301 for patients with PKD is being initiated. The trial is expected to enroll 10 patients between the ages of 8 and 55 years of age with a confirmed PKLR gene mutation and documented anemia who have undergone a prior splenectomy at the following sites:
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.