Skip to main content
OpenTrials
Completed

NCT Number: NCT01863758

Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Severe Haemophilia 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

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Medical University Vienna, Vienna, Austria

Loading trial locations.

About this study

There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Severe haemophilia A (FVIII:C < 1%) according to medical history.
  • Male patients ≥ 18 years old.
  • Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).
  • Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.
  • Immunocompetence (CD4+ count > 200/microliter).
  • HIV-negative, if positive, viral load < 200 particles/microliter or < 400,000 copies/mL.
  • Freely given written informed consent

Exclusion criteria

  • Any coagulation disorder other than haemophilia A.
  • Present or past FVIII inhibitor activity (> 0.6 Bethesda Unit [BU])
  • Severe liver or kidney disease.

Treatment and study plan

Human-cl rhFVIII

Biological

Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection.

Primary outcomes

  1. Annualized Number of Bleeding Episodes (BE) in Phase II

    Time frame: Beginning to the end of Phase II (6 months)

    The annualized number of total BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).

Secondary outcomes

  1. Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II

    Time frame: Beginning to the end of Phase II (6 months)

    The annualized number of spontaneous BEs was calculated for each participant as follows: d*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included.

  2. Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week

    Time frame: Beginning to the end of Phase II (6 months)

    The annualized number of BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included.

  3. Median Dosing Interval During Individually Tailored Prophylaxis

    Time frame: Beginning to the end of Phase II (6 months)

    The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient

  4. Dosage Per Week in Phase II

    Time frame: Beginning to the end of Phase II (6 months)

    The mean dosage per week during Phase II of the study are reported.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
May 29, 2013
Registry last updated
Jan 30, 2018

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.