Background: Primary immunodeficiencies (PIDs) are a group of rare congenital diseases caused by a random mutation in an immune system gene. Smooth Drug Development was involved in the organization of a phase III study to evaluate the efficacy and safety of a drug aimed at treating patients with PIDs.
About a trial: We were in charge of obtaining the Ministry of Health approval for the clinical trial, importation of the drug, selection of the centers, monitoring, data management and final report preparetion. We also supervised the activities of the central laboratory and the logistics of the biosamples. 25 subjects and 4 medical centers were included in the study.
Challenges: There were no difficulties during the study thanks to our team which provided very personal approach to the site teams. At the planning stage of the project, we took into account the rarity of the disease, as well as the great competition among companies conducting research in this nosology.