Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

December 21, 2020 updated by: Octapharma

Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A

Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

110

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belarus
      • Minsk, Belarus
        • Republican Scientific Practical Center for Pediatric Oncology and Hematology
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G2V2
        • University of Alberta
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • BC Children's Hospital
    • Ontario
      • Hamilton, Ontario, Canada, L8S4K1
        • Mc Master Children's Hospital
      • Toronto, Ontario, Canada, M5G 1X8
        • Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Hôpital Ste-Justine
  • France
      • Caen, France
        • L'hôpital Côte de Nacre - CHU de Caen
      • Le Kremlin-Bicêtre, France
        • Centre de traitement de l'hemophilie, Hôpital Bicêtre
      • Marseille, France
        • Hopital de la Timone
      • Nantes, France
        • Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
      • Paris, France
        • Hopital Necker
      • Rennes, France
        • CHU de Rennes - Hôpital Pontchaillou
      • Tours, France
        • Hôpital Trousseau - CHU Tours
  • Georgia
      • Tbilisi, Georgia
        • Institute of Haematology and Transfusiology
  • Germany
      • Bonn, Germany
        • Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
      • Frankfurt, Germany, 60590
        • University Hospital Frankfurt/M
      • Mainz, Germany
        • Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
  • India
      • Pune, India
        • Sahyadri Speciality Hospital, Haematology & BMT Unit
      • Vellore, India
        • Christian Medical College & Hospital, Dept of Haematology
    • Karnataka
      • Manipala, Karnataka, India
        • Kasturba Medical College, Dr. TMA Pai Hospital
  • Italy
      • Perugia, Italy
        • Univ. Di Perugia
      • Torino, Italy
        • Centro di Referimento per le Malattie Emorragiche e Trombotiche
  • Moldova, Republic of
      • Chisinau, Moldova, Republic of
        • Scientific Research Institute of Mother and Child Health Care
  • Morocco
      • Rabat, Morocco
        • Centre Hospitalier Ibn Sina
  • Poland
      • Warsaw, Poland, 00-576
        • University Medical School Warsaw
  • Portugal
      • Porto, Portugal
        • HSJ - Hospital de São João, EPE
  • Russian Federation
      • Moscow, Russian Federation
        • Morozovsky Children's Hospital
  • Slovenia
      • Ljubljana, Slovenia
        • Haemophilia Centre, University Clinical Centre
  • Spain
      • Barcelona, Spain
        • Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
      • Madrid, Spain
        • Hospital Universitario La Paz
  • Ukraine
      • Kiev, Ukraine
        • National Children's Specialized Hospital "OHMATDET"
      • Lviv, Ukraine
        • Institute of blood pathology and transfusion medicine
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Cambridge University Hospital
      • London, United Kingdom, WC1N3JH
        • Great Ormond Street Hospital for Children
  • United States
    • California
      • Sacramento, California, United States, 95817
        • UC Davis
    • Florida
      • Saint Petersburg, Florida, United States, 33701
        • All Children's Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Harvard Children's Hospital Boston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Male patients
  • Severe Hemophilia A (FVIII:C <1%)
  • No previous treatment with FVIII concentrates or other blood products containing FVIII

Exclusion Criteria:

  • Diagnosis with a coagulation disorder other than Hemophilia A
  • Severe liver or kidney disease
  • Concomitant treatment with any systemic immunosuppressive drug

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Human cl rhFVIII
Biological: Human cl rhFVIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
Time Frame: maximum 5 years (100 exposure days)
The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.
maximum 5 years (100 exposure days)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Spontaneous Break-through Bleeds
Time Frame: Maximum 5 years (100 exposure days)
The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
Maximum 5 years (100 exposure days)
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Time Frame: Maximum 5 years (100 exposure days)
A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).
Maximum 5 years (100 exposure days)
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
Time Frame: Maximum 5 years (100 exposure days)
An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none).
Maximum 5 years (100 exposure days)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Occurrence of Any Adverse Event (AE)
Time Frame: 5 years
The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Octapharma

Investigators

  • Study Director: Sigurd Knaub, Octapharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2013

Primary Completion (ACTUAL)

December 14, 2018

Study Completion (ACTUAL)

December 20, 2019

Study Registration Dates

First Submitted

October 18, 2012

First Submitted That Met QC Criteria

October 19, 2012

First Posted (ESTIMATE)

October 23, 2012

Study Record Updates

Last Update Posted (ACTUAL)

January 19, 2021

Last Update Submitted That Met QC Criteria

December 21, 2020

Last Verified

December 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GENA-05
  • 2012-002554-23 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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