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Completed

NCT Number: NCT01712438

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

Investigate 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

Republican Scientific Practical Center for Pediatric Oncology and Hematology, Minsk, Belarus

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Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

Human cl rhFVIII

Biological

Primary outcomes

  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 outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A

Important dates

Study start
2013
Primary completion
2018
Study completion
2019
First posted
Oct 23, 2012
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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