Phase 3
N=15
Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin in Paediatric Subjects With Fibrinogen Deficiency
Congenital Fibrinogen Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02408484 ↗Enrolled (actual)
15
Serious AEs
7.1%
Results posted
Jun 2020
Primary outcome: Primary: 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 — 5; 6; 1; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Octafibrin (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Jun 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
5; 6; 1; 2; 1; 0 | — |
| PRIMARY 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 |
6; 8; 2; 0 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Area Under the Concentration-time Curve Normalised (AUCnorm) |
1.419 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Response - Incremental in Vivo Recovery (IVR) |
1.592 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Terminal Elimination Half-life (t1/2) |
84.356 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Maximum Plasma Concentration (Cmax) |
1.559 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Time to Reach Maximum Plasma Concentration (Tmax) |
1.154 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Mean Residence Time (MRT) |
114.332 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Volume of Distribution (Vss) |
81.651 | — |
| SECONDARY Single-dose Pharmacokinetics of Octafibrin: Clearance (Cl) |
0.756 | — |
| SECONDARY Change in Maximum Clot Firmness (MCF) for the First Bleeding Episode for Each Patients and for All Bleeding Episodes |
3.1; 3.3 | 0.0028 sig |
| SECONDARY Change in the Fibrinogen Level for All Bleeding Episodes up to 1 Hour-post Infusion for the First Bleeding Episode and All Bleeding Episodes |
98.9; 98.1 | — |
| SECONDARY Incremental in Vivo Recovery Following the First Infusion of Octafibrin Administration for the Treatment of the First Bleeding Episode and of All Bleeding Episodes |
1.5; 1.5 | — |
| SECONDARY Efficacy of Octafibrin in All Bleeding Episodes Based on a Four-point Haemostatic Efficacy Scale |
7; 8; 1; 2; 1; 0 | — |
| SECONDARY Efficacy of Octafibrin in All Bleeding Episodes Based on a Two-point Haemostatic Efficacy Scale |
8; 10; 2; 0 | — |
| SECONDARY Efficacy of Octafibrin in Surgical Prophylaxis Based on a Four-point Haemostatic Efficacy Scale |
3; 3; 0; 0; 0; 0 | — |
| SECONDARY Efficacy of Octafibrin in Surgical Prophylaxis Based on a Two-point Haemostatic Efficacy Scale |
3; 3; 0; 0 | — |
| SECONDARY Patients With Elevated Values of Prothrombin Fragments 1+2 |
3 | — |
| SECONDARY Safety Assessment: Immunogenicity Testing for Anti-fibrinogen Antibodies |
2 | — |
| SECONDARY Safety Assessment: Adverse Events |
10 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Aged 200 particles/μL or >400,000 copies/mL.
- Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery.
- Diagnosis or suspicion of a neutralizing anti-fibrinogen inhibitor currently or any time in the past.
- Acute or chronic medical condition which may, in the opinion of investigator, affect the conduct of the study, including subjects receiving immune-modulating drugs (other than anti-retroviral chemotherapy), such as alpha-interferon, predni-sone (equivalent to >10 mg/day), or similar drugs, at study start.
- Treatment with IMP in another interventional clinical study currently or during the past 4 weeks.
Data sourced from ClinicalTrials.gov (NCT02408484). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.