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

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. Identifier: NCT01712438
Recruitment Status : Completed
First Posted : October 23, 2012
Results First Posted : October 21, 2019
Last Update Posted : January 19, 2021
Information provided by (Responsible Party):

Brief Summary:
Investigate 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 110 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
Study Start Date : February 2013
Actual Primary Completion Date : December 14, 2018
Actual Study Completion Date : December 20, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Chlorine

Arm Intervention/treatment
Experimental: Human cl rhFVIII Biological: Human cl rhFVIII

Primary Outcome Measures :
  1. Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors [ Time Frame: maximum 5 years (100 exposure days) ]
    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.

Secondary Outcome Measures :
  1. Frequency of Spontaneous Break-through Bleeds [ Time Frame: Maximum 5 years (100 exposure days) ]
    The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII

  2. Efficacy of Human-cl rhFVIII for the Treatment of Bleeds [ Time Frame: Maximum 5 years (100 exposure days) ]
    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).

  3. Efficacy of Human-cl rhFVIII for Surgical Prophylaxis [ Time Frame: Maximum 5 years (100 exposure days) ]
    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).

Other Outcome Measures:
  1. The Occurrence of Any Adverse Event (AE) [ Time Frame: 5 years ]
    The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.

Information from the National Library of Medicine

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

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

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 identifier (NCT number): NCT01712438

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Sponsors and Collaborators
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Study Director: Sigurd Knaub Octapharma
  Study Documents (Full-Text)

Documents provided by Octapharma:
Study Protocol  [PDF] February 1, 2018
Statistical Analysis Plan  [PDF] April 1, 2019

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Responsible Party: Octapharma Identifier: NCT01712438    
Other Study ID Numbers: GENA-05
2012-002554-23 ( EudraCT Number )
First Posted: October 23, 2012    Key Record Dates
Results First Posted: October 21, 2019
Last Update Posted: January 19, 2021
Last Verified: December 2020
Keywords provided by Octapharma:
Previously untreated patients
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