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

Phase 3

Completed

• Conditions: Severe Hemophilia A
• Interventions: Biological: Human cl rhFVIII

• Registration Number: NCT01712438
• Lead Sponsor: Octapharma

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-10-18
• Study First Submitted QC: 2012-10-19
• Study First Posted: 2012-10-23
• Study Start: 2013-02
• Primary Completion: 2018-12-14
• Results First Submitted: 2019-09-25
• Results First Submitted QC: 2019-10-18
• Results First Posted: 2019-10-21
• Study Completion: 2019-12-20
• Status Verified: 2020-12
• Last Update Submitted: 2020-12-21
• Last Update Posted: 2021-01-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 110

Inclusion Criteria

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Human cl rhFVIII	Human cl rhFVIII	-

Primary Outcome Measures

Name	Time	Method
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors	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.

Secondary Outcome Measures

Name	Time	Method
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis	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).
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	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).
Frequency of Spontaneous Break-through Bleeds	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

Trial Locations

Locations (37)

Republican Scientific Practical Center for Pediatric Oncology and Hematology; 🇧🇾 Minsk, Belarus

Hopital Ste-Justine; 🇨🇦 Montreal, Quebec, Canada

Kasturba Medical College, Dr. TMA Pai Hospital; 🇮🇳 Manipala, Karnataka, India

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

Sahyadri Speciality Hospital, Haematology & BMT Unit; 🇮🇳 Pune, India

Morozovsky Children's Hospital; 🇷🇺 Moscow, Russian Federation

Christian Medical College & Hospital, Dept of Haematology; 🇮🇳 Vellore, India

All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States

UC Davis; 🇺🇸 Sacramento, California, United States

Harvard Children's Hospital Boston; 🇺🇸 Boston, Massachusetts, United States

BC Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

Mc Master Children's Hospital; 🇨🇦 Hamilton, Ontario, Canada

L'hôpital Côte de Nacre - CHU de Caen; 🇫🇷 Caen, France

Centre de traitement de l'hemophilie, Hôpital Bicêtre; 🇫🇷 Le Kremlin-Bicêtre, France

Hopital de la Timone; 🇫🇷 Marseille, France

Hopital Trousseau - CHU Tours; 🇫🇷 Tours, France

Hôpital Necker; 🇫🇷 Paris, France

CHU de Rennes - Hôpital Pontchaillou; 🇫🇷 Rennes, France

University Hospital Frankfurt/M; 🇩🇪 Frankfurt, Germany

Institute of Haematology and Transfusiology; 🇬🇪 Tbilisi, Georgia

Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie; 🇫🇷 Nantes, France

Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT); 🇩🇪 Bonn, Germany

Universitätsmedizin der Johannes-Gutenberg-Universität Mainz; 🇩🇪 Mainz, Germany

Univ. Di Perugia; 🇮🇹 Perugia, Italy

Scientific Research Institute of Mother and Child Health Care; 🇲🇩 Chisinau, Moldova, Republic of

Centre Hospitalier Ibn Sina; 🇲🇦 Rabat, Morocco

University Medical School Warsaw; 🇵🇱 Warsaw, Poland

Centro di Referimento per le Malattie Emorragiche e Trombotiche; 🇮🇹 Torino, Italy

HSJ - Hospital de São João, EPE; 🇵🇹 Porto, Portugal

Haemophilia Centre, University Clinical Centre; 🇸🇮 Ljubljana, Slovenia

Unitat d'hemofilia, Hospital Universitari Vall d'Hebron; 🇪🇸 Barcelona, Spain

National Children's Specialized Hospital "OHMATDET"; 🇺🇦 Kiev, Ukraine

Institute of Blood Pathology and Transfusion Medicine; 🇺🇦 Lviv, Ukraine

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom

Cambridge University Hospital; 🇬🇧 Cambridge, United Kingdom

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain