NCT02408484

Prospective, Open-label, Uncontrolled, Phase III Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery in Paediatric Subjects With Congenital Fibrinogen Deficiency

Completed Phase 3 Results posted Last updated 21 December 2020

What this trial tests

Phase 3 trial testing Octafibrin in Congenital Fibrinogen Deficiency in 15 participants. Completed in 11 June 2019.

Timeline

1 December 2015

Primary endpoint
11 June 2019

11 June 2019

Quick facts

Lead sponsor	Octapharma
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	15
Start date	1 December 2015
Primary completion	11 June 2019
Estimated completion	11 June 2019
Sites	4 locations across India, Iran, Lebanon

Drugs / interventions tested

• Octafibrin — full drug profile →

Conditions studied

• Congenital Fibrinogen Deficiency — all drugs for Congenital Fibrinogen Deficiency →

Sponsor

Octapharma — full company profile →

Who can join

Under 11, any sex, with Congenital Fibrinogen Deficiency. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in the On-demand Treatment of the First Documented Bleeding Episode of Each Patient Based on a 4-point Haemostatic Efficacy Scale
  Time frame: First Octafibrin infusion for the treatment of a bleeding episode until 24 hours (i.e., 1 day) after the last infusion or the end of the treatment observation period, whichever comes last
  The overall clinical assessment of the haemostatic efficacy of Octafibrin in treating the first documented bleeding episode (BE) of each patient. The first bleeding episode covered the time from the first Octafibrin infusion until 24 hours (i.e. 1 day) after the last infusion or the end of the treatment observation period, which ever came last. The investigator's overall clinical assessment of ha
• Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in the On-demand Treatment of the First Documented Bleeding Episode of Each Patient Based on a 2-point Haemostatic Efficacy Scale
  Time frame: From the first Octafibrin infusion until 24 hours (i.e. 1 day) after the last infusion or the end of the treatment observation period, which ever came last.
  The overall clinical assessment of the haemostatic efficacy of Octafibrin in treating the first documented bleeding episode (BE) of each patient. The first bleeding episode covered the time from the first Octafibrin infusion until 24 hours (i.e. 1 day) after the last infusion or the end of the treatment observation period, which ever came last. The investigator's overall clinical assessment of ha

Sponsor's own description

This study will assess the efficacy of Octafibrin, a fibrinogen concentrate in in the on-demand treatment of spontaneous or traumatic bleeding episodes in paediatric patients less than 12 years of age.The planned study duration is up to 5 years. The study will be considered completed when a minimum of 6 subjects (i.e., at least 3 subjects aged between 0 and \<6 years and 3 subjects aged between 6 and \<12 years) have at least one documented bleeding episode and when in total a minimum of 2 surgical procedures have been performed.

All patients will undergo a pharmacokinetic (PK) study after screening. This will have a duration of 14 days, after which a patient can be treated for a bleeding episode or planned surgical procedure when they occur.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Efficacy and safety of fibrinogen concentrate for on-demand treatment of bleeding and surgical prophylaxis in paediatric patients with congenital fibrinogen deficiency.
   Djambas Khayat C, Lohade S, D'Souza F, Shamanur LG, et al · · 2021 · cited 11× · PMID 33326665 · DOI 10.1111/hae.14230
2. Efficacy and safety of fibrinogen concentrate for perioperative prophylaxis of bleeding in adult, adolescent, and pediatric patients with congenital fibrinogen deficiency: FORMA-02 and FORMA-04 clinical trials.
   Khayat CD, Lohade S, Zekavat OR, Kruzhkova I, et al · · 2022 · cited 2× · PMID 35932202 · DOI 10.1111/trf.17029