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

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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

Tracking Information
First Submitted Date  ICMJE November 19, 2013
First Posted Date  ICMJE November 25, 2013
Results First Submitted Date  ICMJE November 27, 2019
Results First Posted Date  ICMJE December 17, 2019
Last Update Posted Date January 19, 2021
Study Start Date  ICMJE April 2014
Actual Primary Completion Date December 27, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 27, 2019)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: November 19, 2013)
Immunogenicity [ Time Frame: Maximum two years ]
Number of patients who develop an inhibitor
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 27, 2019)
  • 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).
  • 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.
  • 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".
  • 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.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Official Title  ICMJE Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Severe Hemophilia A
Intervention  ICMJE Biological: Human-cl rhFVIII
Study Arms  ICMJE Experimental: Human-cl rhFVIII
Intervention: Biological: Human-cl rhFVIII
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 26, 2019)
48
Original Estimated Enrollment  ICMJE
 (submitted: November 19, 2013)
100
Actual Study Completion Date  ICMJE December 27, 2018
Actual Primary Completion Date December 27, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   France,   Georgia,   India,   Moldova, Republic of,   Poland,   Ukraine,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01992549
Other Study ID Numbers  ICMJE GENA-15
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Octapharma
Study Sponsor  ICMJE Octapharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Sigurd Knaub, PhD Octapharma
PRS Account Octapharma
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP