Introduction:

Thymic hypoplasia (TH) is a disease classified as an inborn error of immunity. To date, there is no registered therapy for it in Cuba or the world.

Objective:

To evaluate the safety of Biomodulina T in children with TH associated or not with cellular immunodeficiency.

Methods:

Phase III, multicenter, uncontrolled clinical trial, which included 60 children between one and five years of age with TH, 44 not associated with cellular immunodeficiency (group I) and 16 associated with cellular immunodeficiency (group II). All received intramuscular Biomodulina T in two four-week cycles separated by four weeks of rest. Doses of one, two or three vials of 3 mg/week were assigned according to the degree of TH. At 16 weeks, those who had not reached the normal size of the thymus received a third cycle of 3 mg weekly for 8 weeks. Safety variables were measured at each administration of the product and throughout the trial.

Results:

Of the total number of children who received at least one dose, 68.3% reported no adverse events (AEs), and 31.7% reported at least one event. The occurrence of AEs was higher in group II. The most frequently reported AEs were injection site reaction, allergic skin reactions, and fever. Most of these were mild, not severe, and had no change in treatment-related attitude.

Conclusions:

Biomodulina T in a three-cycle treatment regimen is safe in children with TH.

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