Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A

NCT01125813 | Completed Phase 3 Results DMC

• 1 other identifier: GENA-08
• Study Type: interventional
• Target: 32
• Locations: 4 countries
• Sites: 11

Brief Summary

This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (2)

• Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months

  Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.

  At least 50 Exposure Days and at least 6 months

• Efficacy of Treating Bleeding Episodes

  At the end of a bleeding episode, efficacy was assessed as: * Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion * Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution * Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution * None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated

  After each bleeding episode, up to 6 month

Study Arms (1)

human cl-rhFVIII

EXPERIMENTAL

Biological: recombinant Factor VIII

Interventions

recombinant Factor VIII BIOLOGICAL

intravenous infusion of factor FVIII every other day.

Also known as: human-cl rhFVIII

human cl-rhFVIII

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Severe hemophilia A ((FVIII:C \<= 1%)
• Male subjects \>= 12 years of age
• Previously treated with FVIII concentrate, at least 50 EDs
• Immunocompetent (CD4+ count \> 200/ul)
• Negative for anti- HIV; if positive, viral load \< 200 particles/u; or \<400,000 copies/mL

You may not qualify if:

• Other coagulation disorder than hemophilia A
• Present of past FVIII inhibitor activity (.= 0.6 BU)
• Severe liver and kidney disease
• Receiving of scheduled to receive immuno-modulating drugs

Sponsors & Collaborators

• Octapharma: lead

Study Sites (11)

Medizinische Universitaet Wien

Vienna, 1090, Austria

Location

Haematological Hospital Joan Pavel

Sofia, 1233, Bulgaria

Location

Werlhof Institut fuer Haemostaseologie GmbH

Hanover, Lower Saxony, 30159, Germany

Location

Universitaetsklinikum

Bonn, Germany

Location

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

SRH Kurpfalzkrankenhaus Heidelberg

Heidelberg, 69123, Germany

Location

Basingstoke & North Hampshire NHS Foundation Trust

Basingstoke, RG24 9NA, United Kingdom

Location

University Hospital of Wales

Cardiff, United Kingdom

Location

Royal Free Hospital

London, United Kingdom

Location

Manchester Royal Infirmary

Manchester, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

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

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Dr Sigurd Knaub
• Organization: Octapharma AG

sigurd.knaub@octapharma.ch

41 55 451 21 41

Study Officials

• Johannes Oldenburg, Prof.

  Universitaetsklinikum Bonn

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• 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 17, 2010
• First Posted: May 18, 2010
• Study Start: June 1, 2010
• Primary Completion: January 1, 2012
• Study Completion: January 1, 2012
• Last Updated: September 11, 2017
• Results First Posted: April 22, 2013

Record last verified: 2017-08

Locations

BU (1); DE (4); AU (1); GB (5)