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

Severe Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01712438
• Other study ID #: GENA-05
• Secondary ID: 2012-002554-23
• Status: Completed
• Phase: Phase 3
• Start date: February 2013
• Est. completion date: December 20, 2019

Study information

• Verified date: December 2020
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 110
• Est. completion date: December 20, 2019
• Est. primary completion date: December 14, 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

Biological:
• Human cl rhFVIII

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

United States,  Belarus,  Canada,  France,  Georgia,  Germany,  India,  Italy,  Moldova, Republic of,  Morocco,  Poland,  Portugal,  Russian Federation,  Slovenia,  Spain,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	The Occurrence of Any Adverse Event (AE)	The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.	5 years
Primary	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	Frequency of Spontaneous Break-through Bleeds	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)
Secondary	Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	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)
Secondary	Efficacy of Human-cl rhFVIII for Surgical Prophylaxis	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)