Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery

NCT02267226 | Completed Phase 3 Results

• 1 other identifier: FORMA-02
• Study Type: interventional
• Target: 25
• Locations: 9 countries
• Sites: 12

Brief Summary

The purpose of the study is to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiency.

Conditions

Congenital Fibrinogen Deficiency

Outcome Measures

Primary Outcomes (1)

• Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in Treating the First Documented Bleeding Episode of Each Patient.

  The first bleeding episode covers the time period from the first Octafibrin infusion until 24 hours (i.e., 1 day) after the last infusion. The investigator's overall clinical assessment of haemostatic efficacy for bleeding was based on a 4 point haemostatic efficacy scale. The final efficacy assessment of each patient was adjudicated by the Independent Data Monitoring \& Endpoint Adjudication Committee (IDMEAC).

  24 hours after last infusion for each bleeding episode

Secondary Outcomes (5)

• Maximum Clot Firmness (MCF) After Fibrinogen Infusion in Each Documented Bleeding Episode (BE), Measured in Frozen Plasma in a Central Laboratory.

  Before first infusion and 1 hour after end of first and last infusion of each documented bleeding episode

• Fibrinogen Plasma Level

  Before (pre-infusion), 1 hour and 3 hours after the end of each subsequent infusion as well as at the time of the overall clinical assessment of haemostatic efficacy (i.e., 24 hours after the last infusion of each documented bleeding episode)

• Response as Indicated by Incremental in Vivo Recovery (IVR)

  Pre-infusion and 1 and 3 hours post-infusion

• Efficacy of Octafibrin for All Bleeding Episodes Collected in the Study

  24 hours after last infusion for each bleeding episode

• Efficacy of Octafibrin in Preventing Bleeding During and After Surgery

  First dose of Octafibrin administered prior to elective surgery to at least 3 post-operative days for minor and 7 post-operative days for major surgeries or last post-operative infusion, whichever comes last

Other Outcomes (1)

• Analysis of Safety: Immunogenicity Testing for Anti-fibrinogen Antibodies

  Up to 30 days (start of the first Octafibrin infusion until the end of each 30-day observation and follow-up period for on-demand treatment or until the Last Post-Operative Day in surgeries)

Study Arms (1)

Octafibrin

EXPERIMENTAL

Drug: Octafibrin

Interventions

Octafibrin DRUG

Octafibrin

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged ≥12 years (only 18 and above in Russia)
• Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis:
• Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia.
• Historical plasma fibrinogen activity of \<50 mg/dL or levels below the limit of detection of the local assay method.
• Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
• Informed consent signed by the subject or legal guardian.

You may not qualify if:

• Life expectancy \<6 months.
• Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia.
• Prophylactic treatment with a fibrinogen concentrate.
• Treatment with:
• Any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the bleeding episode or surgery.
• Any coagulation-active drug (i.e., non-steroidal anti-inflammatory drugs, warfarin, coumarin derivatives, platelet aggregation inhibitors) within 1 week prior to start of treatment for the bleeding episode or surgery, or as a planned or expected medication during the time period from Day 1 until 24 hours (i.e., 1 day) after the last Octafibrin infusion.
• Presence or history of:
• Hypersensitivity to study medication.
• Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment for the bleeding episode or surgery.
• Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or surgery
• Hypersensitivity to human plasma proteins.
• Oesophageal varicose bleeding.
• End-stage liver disease (i.e., Child-Pugh score B or C).
• Pregnant women within the first 20 weeks of gestation.
• Currently breast-feeding.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (12)

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Dept of Clinical Hematology for Hemorrhagic Diatheses and Anaemia, SHAT "Joan Pavel"

Sofia, Bulgaria

Location

St. John's Medical College Hospital

Bangalore, India

Location

Sahyadri Specialty Hospital

Pune, India

Location

Dept of Hematology, Christian Medical College

Vellore, India

Location

Seyed Al Shohada Hospital

Isfahan, Iran

Location

Dastgheib Hospital

Shīrāz, Iran

Location

Hotel-Dieu de France

Beirut, Lebanon

Location

Haematological Scientific Center of Ministry of Healthcare of the Russian Federation

Moscow, Russia

Location

Centre of Excellence in Thrombosis & Hemostasis, King Saud University

Riyadh, Saudi Arabia

Location

Dept of Haematology, Ege University Children's Hospital

Izmir, Turkey (Türkiye)

Location

Centre for Haemostasis & Thrombosis, St Thomas' Hospital

London, United Kingdom

Location

Results Point of Contact

• Title: Director of Clinical Operations
• Organization: Octapharma USA

sylvia.werner@octapharma.com

201 604-1149

Study Officials

• Cristina Solomon, MD

  Octapharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2014
• First Posted: October 17, 2014
• Study Start: September 1, 2014
• Primary Completion: February 14, 2018
• Study Completion: February 14, 2018
• Last Updated: January 15, 2021
• Results First Posted: February 20, 2019

Record last verified: 2020-12

Locations

BU (1); US (1); LE (1); RU (1); TU (1); IN (3); SA (1); IR (2); GB (1)