Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

NCT01863758 | Completed Phase 3 Results DMC

• 1 other identifier: GENA-21
• Study Type: interventional
• Target: 66
• Locations: 8 countries
• Sites: 20

Brief Summary

To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.

Conditions

Severe Haemophilia A

Outcome Measures

Primary Outcomes (1)

• Annualized Number of Bleeding Episodes (BE) in Phase II

  The annualized number of total BEs was calculated for each participant as follows: d\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).

  Beginning to the end of Phase II (6 months)

Secondary Outcomes (4)

• Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II

  Beginning to the end of Phase II (6 months)

• Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week

  Beginning to the end of Phase II (6 months)

• Median Dosing Interval During Individually Tailored Prophylaxis

  Beginning to the end of Phase II (6 months)

• Dosage Per Week in Phase II

  Beginning to the end of Phase II (6 months)

Study Arms (1)

Human-cl rhFVIII

EXPERIMENTAL

Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.

Biological: Human-cl rhFVIII

Interventions

Human-cl rhFVIII BIOLOGICAL

Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection.

Human-cl rhFVIII

Eligibility Criteria

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

You may qualify if:

• Severe haemophilia A (FVIII:C \< 1%) according to medical history.
• Male patients ≥ 18 years old.
• Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).
• Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.
• Immunocompetence (CD4+ count \> 200/microliter).
• HIV-negative, if positive, viral load \< 200 particles/microliter or \< 400,000 copies/mL.
• Freely given written informed consent

You may not qualify if:

• Any coagulation disorder other than haemophilia A.
• Present or past FVIII inhibitor activity (\> 0.6 Bethesda Unit \[BU\])
• Severe liver or kidney disease.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (20)

Medical University Vienna

Vienna, Austria

Location

University Multiprofile Hospital for Active Treatment

Plovdiv, Bulgaria

Location

Specialized Hospital for Active Treatment

Sofia, Bulgaria

Location

Multiprofile Hospital for Active Treatment

Varna, Bulgaria

Location

Vivantes Hospital in Friedrichshain

Berlin, Germany

Location

SRH Kurpfalzklinik Heidelberg GMBH

Heidelberg, Germany

Location

Hungarian National Healthcare Center

Budapest, Hungary

Location

University of Debrecen, Medical and Health Science Center

Debrecen, Hungary

Location

University Teaching Hospital in Bialystok, Teaching Department of Hematology with a Subdepartment of Vascular Diseases

Bialystok, Poland

Location

University Clinical Center, Teaching Department of Hematology and Transplantology

Gdansk, Poland

Location

Nicolaus Copernicus Municipal Specialist Hospital, Department of Hematology

Torun, Poland

Location

Institute of Hematology and Transfusion Medicine, Depart. of Hemostatic Disorders and Internal Diseases

Warsaw, Poland

Location

Sanador SRL

Bucharest, Romania

Location

Louis Turcanu Emergency Clinical Children's Hospital

Timișoara, Romania

Location

University Hospital Saint Cyril and Metod

Bratislava, Slovakia

Location

University Hospital Martin, Department of Hematology and Transfusiology

Martin, Slovakia

Location

Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center

Basingstoke, United Kingdom

Location

Royal London Hospital, Barts and the London Hemophilia Center

London, United Kingdom

Location

Manchester Royal Infirmary, Department of Clinical Hematology

Manchester, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, 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

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: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2013
• First Posted: May 29, 2013
• Study Start: August 1, 2013
• Primary Completion: January 1, 2015
• Study Completion: January 1, 2015
• Last Updated: January 30, 2018
• Results First Posted: January 30, 2018

Record last verified: 2017-07

Data Sharing

• IPD Sharing: Will not share

Locations

GB (4); SL (2); AU (1); PL (4); DE (2); BU (3); HU (2); RO (2)