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

NCT01712438 | Completed Phase 3 Results DMC

• 2 other identifiers: GENA-052012-002554-23
• Study Type: interventional
• Target: 110
• Locations: 16 countries
• Sites: 36

Brief Summary

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

Conditions

Severe Hemophilia A

Keywords

Previously untreated patients

Outcome Measures

Primary Outcomes (1)

• Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors

  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 Outcomes (3)

• Frequency of Spontaneous Break-through Bleeds

  Maximum 5 years (100 exposure days)

• Efficacy of Human-cl rhFVIII for the Treatment of Bleeds

  Maximum 5 years (100 exposure days)

• Efficacy of Human-cl rhFVIII for Surgical Prophylaxis

  Maximum 5 years (100 exposure days)

Other Outcomes (1)

• The Occurrence of Any Adverse Event (AE)

  5 years

Study Arms (1)

Human cl rhFVIII

EXPERIMENTAL

Biological: Human cl rhFVIII

Interventions

Human cl rhFVIII BIOLOGICAL

Human cl rhFVIII

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (37)

UC Davis

Sacramento, California, 95817, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Harvard Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Republican Scientific Practical Center for Pediatric Oncology and Hematology

Minsk, Belarus

Location

University of Alberta

Edmonton, Alberta, T6G2V2, Canada

Location

BC Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

Mc Master Children's Hospital

Hamilton, Ontario, L8S4K1, Canada

Location

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Hopital Ste-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

L'hôpital Côte de Nacre - CHU de Caen

Caen, France

Location

Centre de traitement de l'hemophilie, Hôpital Bicêtre

Le Kremlin-Bicêtre, France

Location

Hopital de la Timone

Marseille, France

Location

Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie

Nantes, France

Location

Hôpital Necker

Paris, France

Location

CHU de Rennes - Hôpital Pontchaillou

Rennes, France

Location

Hopital Trousseau - CHU Tours

Tours, France

Location

Institute of Haematology and Transfusiology

Tbilisi, Georgia

Location

Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)

Bonn, Germany

Location

University Hospital Frankfurt/M

Frankfurt, 60590, Germany

Location

Universitätsmedizin der Johannes-Gutenberg-Universität Mainz

Mainz, Germany

Location

Kasturba Medical College, Dr. TMA Pai Hospital

Manipal, Karnataka, India

Location

Sahyadri Speciality Hospital, Haematology & BMT Unit

Pune, India

Location

Christian Medical College & Hospital, Dept of Haematology

Vellore, India

Location

Univ. Di Perugia

Perugia, Italy

Location

Centro di Referimento per le Malattie Emorragiche e Trombotiche

Torino, Italy

Location

Scientific Research Institute of Mother and Child Health Care

Chisinau, Moldova

Location

Centre Hospitalier Ibn Sina

Rabat, Morocco

Location

University Medical School Warsaw

Warsaw, 00-576, Poland

Location

HSJ - Hospital de São João, EPE

Porto, Portugal

Location

Morozovsky Children's Hospital

Moscow, Russia

Location

Haemophilia Centre, University Clinical Centre

Ljubljana, Slovenia

Location

Unitat d'hemofilia, Hospital Universitari Vall d'Hebron

Barcelona, Spain

Location

Hospital Universitario La Paz

Madrid, Spain

Location

National Children's Specialized Hospital "OHMATDET"

Kiev, Ukraine

Location

Institute of Blood Pathology and Transfusion Medicine

Lviv, Ukraine

Location

Cambridge University Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N3JH, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Sylvia Werner
• Organization: Octapharma

sylvia.werner@octapharma.com

415 260-9577

Study Officials

• Sigurd Knaub

  Octapharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

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: October 18, 2012
• First Posted: October 23, 2012
• Study Start: February 1, 2013
• Primary Completion: December 14, 2018
• Study Completion: December 20, 2019
• Last Updated: January 19, 2021
• Results First Posted: October 21, 2019

Record last verified: 2020-12

Locations

MO (1); IN (3); UA (2); DE (3); US (3); PT (1); ES (2); GB (2); PL (1); BE (1); CA (5); FR (7); RU (1); SL (1); IT (2); GE (1)