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Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01992549
Recruitment Status : Completed
First Posted : November 25, 2013
Results First Posted : December 17, 2019
Last Update Posted : January 19, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Description
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Brief Summary:
The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

Condition or disease Intervention/treatment Phase
Severe Hemophilia A Biological: Human-cl rhFVIII Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Study Start Date : April 2014
Actual Primary Completion Date : December 27, 2018
Actual Study Completion Date : December 27, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Chlorine

Arms and Interventions
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Arm Intervention/treatment
Experimental: Human-cl rhFVIII Biological: Human-cl rhFVIII


Outcome Measures
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Primary Outcome Measures :
  1. Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors [ Time Frame: Maximum two years ]
    The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.


Secondary Outcome Measures :
  1. Frequency of Spontaneous Break-through Bleeds [ Time Frame: Maximum 2 years ]
    The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).

  2. Efficacy of Human-cl rhFVIII for the Treatment of Bleeds [ Time Frame: Maximum 2 years ]
    A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.

  3. Efficacy of Human-cl rhFVIII for Surgical Prophylaxis [ Time Frame: Maximum 2 years ]
    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). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".

  4. The Occurrence of Any Adverse Event (AE) [ Time Frame: Maximum 2 years ]
    The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

1. Patients who completed GENA-05 in accordance with the study protocol

Exclusion Criteria:

  1. Severe liver or kidney disease
  2. Concomitant treatment with any systemic immunosuppressive drug;
  3. Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01992549


Locations
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United States, California
UC Davis Medical Center
Sacramento, California, United States, 95817
Canada, Alberta
University of Alberta
Edmonton, Alberta, Canada
Canada, British Columbia
BC Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Ontario
McMaster Children's Hospital
Hamilton, Ontario, Canada, L8S4K1
Canada
Hospital for Sick Children
Toronto, Canada
France
Hopital de la Timone
Marseille, France
Hôpital Kremlin Bicètre
Paris, France
Georgia
Institute of Hematology and Transfusiology
Tbilisi, Georgia
India
Sahyadri Speciality Hospital
Pune, India, 411004
Christian Medical College
Vellore, India, 632004
Moldova, Republic of
IMSP Mother and Child Institute
Chişinău, Moldova, Republic of
Poland
University Medical School
Warsaw, Poland
Ukraine
The National Children Specialized Hospital "OHMATDET"
Kiev, Ukraine
Danylo Halytsky Lviv National Medical University
Lviv, Ukraine
United Kingdom
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Octapharma
Investigators
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Study Director: Sigurd Knaub, PhD Octapharma
  Study Documents (Full-Text)

Documents provided by Octapharma:
Study Protocol  [PDF] September 18, 2013
Statistical Analysis Plan  [PDF] April 30, 2019

More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01992549    
Other Study ID Numbers: GENA-15
First Posted: November 25, 2013    Key Record Dates
Results First Posted: December 17, 2019
Last Update Posted: January 19, 2021
Last Verified: December 2020
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn