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Completed

NCT Number: NCT01992549

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

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.

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Key information

Conditions

Severe Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

University of Alberta, Edmonton, Alberta, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • Severe liver or kidney disease
  • Concomitant treatment with any systemic immunosuppressive drug;
  • Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Treatment and study plan

Human-cl rhFVIII

Biological

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Important dates

Study start
2014
Primary completion
2018
Study completion
2018
First posted
Nov 25, 2013
Registry last updated
Jan 19, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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