FORMA 04 Trial

FORMA-04 was a multinational, multicenter, prospective, open-label, uncontrolled Phase 3 study designed to evaluate the haemostatic efficacy of fibryga® in the on-demand treatment of acute bleeding episodes (BE) and surgical prophylaxis in pediatric patients (0-12 years) with congenital fibrinogen deficiency (CFD).

The study included 11 patients diagnosed with afibrinogenaemia, all of whom completed the trial.

Primary objective

To assess haemostatic efficacy for on-demand treatment of bleeding and surgical prophylaxis using a standardized 4-point efficacy scale: excellent, good, moderate, or none.

Table 1: Efficacy assessment for treatment of BEs (Bleeding population, N = 10 BEs in eight patients)

Table 2: Intraoperative and postoperative evaluation of fibryga^® efficacy in surgical prophylaxis

Safety

Ten AEs occurred in four patients. Two AEs were deemed possibly related to treatment. which both occurred in the same patient. One of them were considered as SAE and led to discontinuation of the patient from the study. There were no clinical signs of neutralizing anti-fibrinogen antibodies in any patients during the study.

Table 3: Summary of AEs (Safety Population, N=14)