Clinical Trials:
Fanconi Anemia
RP-L102 is being developed for FA, a rare DNA repair disorder characterized by bone marrow failure, a predisposition to cancer and often death before the age of 30.
An open-label, global registrational clinical trial of RP-L102 for the treatment of FA is currently ongoing. The trial aims to enroll 12 patients total from the U.S. and EU. The primary endpoint of the trial, which has been agreed upon by the FDA and EMA is mitomycin-C (MMC) resistance, a measure of bone marrow cell correction.
For more information about FA, visit our Patients and Community 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.