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    FORMA 02 Trial

    FORMA 02 Trial

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    FORMA-02 was a prospective, open-label, uncontrolled phase III study designed to evaluate the efficacy and safety of fibryga® for on-demand treatment of acute bleeding and for bleeding prevention during and after surgery in patients aged 12 years and older with congenital fibrinogen deficiency (afibrinogenemia).

    fibryga® was dosed individually to achieve recommended target fibrinogen plasma levels, tailored to the severity of the bleeding (minor or major) or the type of surgery (minor or major).

    In total, 24 patients experienced a bleeding episode, while nine underwent surgery. The safety population included 25 patients overall.

    Primary objective

    To demonstrate the efficacy of fibryga® for on-demand treatment of acute bleeding episodes (spontaneous or after trauma).

    Efficacy

    Haemostatic efficacy for on-demand treatment of bleeding

    Treatment success (rating of excellent or good) was 96.6% (90% CI: 0.92–0.99) according to the Investigator, and 98.9% (90% CI: 0.95–1.0) according to the Independent Data Monitoring and Endpoint Adjudication Committee (IDMEAC)

    Maximum Clot Firmness

    Haemostatic efficacy was shown by a significant rise in mean (± SD) MCF from 0 mm at baseline to 5.79 (± 2.53) mm (p < 0.001) at 1 hour post-fibryga® infusion. The overall mean (± SD) increase in MCF was 6.48 (± 3.07) mm.

    Haemostatic efficacy for surgical prophylaxis

    The intra-operative and post-operative efficacy of fibryga® was rated as 100% (90% CI: 0.82–1.00) by the investigator, either the surgeon or haematologist, as well as the IDMEAC.

    Haemostatic efficacy for on-demand treatment of bleeding

    A total of 89 bleeding episodes occurred during the study; 87 were considered minor and two were considered major. Most bleeding episodes (83/89; 93.3%) required a single infusion of fibryga®, with a mean (± SD) dose of 61.88 (± 11.73) mg/kg for the first infusion.

    Table 1: Efficacy of fibryga® for on-demand treatment of bleeding

    Haemostatic efficacy for surgical prophylaxis.

    Nine patients received fibryga® during 12 surgeries (11 were minor surgeries). The mean (± SD) pre-operative loading dose was 77.39 (± 20.22) mg/kg (11 surgeries) and the mean (± SD) maintenance dose was 20.13 (± 7.49) mg/kg.

    Table 2: Efficacy of fibryga® for surgical prophylaxis

    Four-Point Efficacy Scale [%] Excellent Good Moderate None Intra-operative Surgeon 91.7 8.3 0 0 IDMEAC 91.7 8.3 0 0 Post-operative Haematologist 100.0 0 0 0 IDMEAC 91.7 8.3 0 0 12 surgeries in nine patients. IDMEAC: Independent Data Monitoring and Endpoint Adjudication Committee. Source: Based on Lissitchkov et al. 2019.
    Four-Point Efficacy Scale [%] Excellent Good Moderate None Intra-operative Surgeon 91.7 8.3 0 0 IDMEAC 91.7 8.3 0 0 Post-operative Haematologist 100.0 0 0 0 IDMEAC 91.7 8.3 0 0 12 surgeries in nine patients. IDMEAC: Independent Data Monitoring and Endpoint Adjudication Committee. Source: Based on Lissitchkov et al. 2019.

    Safety

    Overall, 91 AEs occurred in 19 patients (76.0%). Fifteen SAEs were observed in five patients. One serious (thrombosis) and two non-serious (drug eruption and phlebitis) AEs were classified as possibly or probably treatment related. No deaths were reported.

    Reference

    Lissitchkov T, Madan B, Djambas Khayat C, Zozulya N, Ross C, Karimi M, Kavakli K, De Angulo GR, Almomen A, Schwartz BA, Solomon C, Knaub S, Peyvandi F. Efficacy and safety of a new human fibrinogen concentrate in patients with congenital fibrinogen deficiency: an interim analysis of a Phase III trial. Transfusion. 2018 Feb;58(2):413-422. doi: 10.1111/trf.14421. Epub 2017 Nov 30. PMID: 29194665. [PubMed]

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