Primary immunodeficiencies (pediatric population)

Background: Primary immunodeficiencies (PIDs) are a group of rare congenital diseases caused by a random mutation in an immune system gene. Smooth Drag Development was responsible for organizing a phase III study to evaluate the efficacy and safety of a drug to treat children with diagnosed primary immunodeficiencies.

About a trial: Our responsibility was to receive the approval of the Ministry of Health, import of the drug, site selection, monitoring, data management and preparation of the final report. Smooth Drug Development was in charge of coordinating a central laboratory. We also handled patient accommodation and logistics. Given the rarity and severity of the disease, the Sponsor provided the subjects and their parents transportation from their place of residence to the city where the sites were located, taxi, hotels and meals. Our company organized this process. 14 subjects at 3 medical centers were enrolled in the study. Recruitment was completed in 3 months. We already had experience in a similar study with adult subjects.

Challenges: In this study, we encountered a previous denial of approval due to the pediatric population. After this project was handed over from another CRO, which received denial, our team together with the Sponsor prepared a new application for the approval of the trial, prepared a detailed scientific rationale for the inclusion subjects under 6 years of age. This time, the MoH granted approval for the trial.

Also in this project, the protocol included many procedures and blood draws. Our team provided thorough training and instructions on the trial for doctors. We also provided freezers to centers that did not have them.

After this study, the Sponsor changed the instructions for use and expanded the age of the patients.