Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (guardian™ 5)

April 24, 2020 updated by: Novo Nordisk A/S

A Multi-centre Non-interventional Study of Safety and Efficacy of Turoctocog Alfa (rFVIII) During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII =<2%)

This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
69

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Graz, Austria, 8036
        • Novo Nordisk Investigational Site
      • Linz, Austria, 4020
        • Novo Nordisk Investigational Site
  • Czechia
      • Brno, Czechia, 625 00
        • Novo Nordisk Investigational Site
  • France
      • Bordeaux, France, 33076
        • Novo Nordisk Investigational Site
      • Montmorency, France, 95160
        • Novo Nordisk Investigational Site
      • Rennes, France, 35033
        • Novo Nordisk Investigational Site
      • Saint Etienne, France, 42055
        • Novo Nordisk Investigational Site
      • Strasbourg, France, 67098
        • Novo Nordisk Investigational Site
  • Germany
      • Braunschweig, Germany, 38118
        • Novo Nordisk Investigational Site
      • Duisburg, Germany, 47051
        • Novo Nordisk Investigational Site
      • Frankfurt / Main, Germany, 60596
        • Novo Nordisk Investigational Site
      • Giessen, Germany, 35392
        • Novo Nordisk Investigational Site
      • Leipzig, Germany, 04289
        • Novo Nordisk Investigational Site
      • Mörfelden-Walldorf, Germany, 64546
        • Novo Nordisk Investigational Site
      • München, Germany, 80331
        • Novo Nordisk Investigational Site
      • Münster, Germany, 48143
        • Novo Nordisk Investigational Site
  • Greece
      • Athens, Greece, GR-11527
        • Novo Nordisk Investigational Site
  • Hungary
      • Budapest, Hungary, H-1134
        • Novo Nordisk Investigational Site
      • Debrecen, Hungary, 4032
        • Novo Nordisk Investigational Site
      • Mohács, Hungary, 7700
        • Novo Nordisk Investigational Site
  • Italy
      • Castelfranco Veneto, Italy, 31033
        • Novo Nordisk Investigational Site
      • Catania, Italy, 95123
        • Novo Nordisk Investigational Site
      • Firenze, Italy, 50134
        • Novo Nordisk Investigational Site
      • Padova, Italy, 35128
        • Novo Nordisk Investigational Site
      • Palermo, Italy, 90127
        • Novo Nordisk Investigational Site
  • Netherlands
      • Den Haag, Netherlands, 2545AA
        • Novo Nordisk Investigational Site
      • Utrecht, Netherlands, 3584 CX
        • Novo Nordisk Investigational Site
  • Poland
      • Warszawa, Poland, 02-091
        • Novo Nordisk Investigational Site
  • Slovakia
      • Banska Bystrica, Slovakia, 975 17
        • Novo Nordisk Investigational Site
      • Kosice, Slovakia, 04001
        • Novo Nordisk Investigational Site
  • Slovenia
      • Ljubljana, Slovenia, 1000
        • Novo Nordisk Investigational Site
  • Spain
      • Barcelona, Spain, 08035
        • Novo Nordisk Investigational Site
  • Sweden
      • Malmö, Sweden, 205 02
        • Novo Nordisk Investigational Site
      • Stockholm, Sweden, 171 76
        • Novo Nordisk Investigational Site
  • Switzerland
      • Zürich, Switzerland, 8091
        • Novo Nordisk Investigational Site
  • United States
    • Alabama
      • Mobile, Alabama, United States, 36604
        • Novo Nordisk Investigational Site
    • California
      • Torrance, California, United States, 90502-2004
        • Novo Nordisk Investigational Site
    • Illinois
      • Peoria, Illinois, United States, 61615
        • Novo Nordisk Investigational Site
    • New Mexico
      • Albuquerque, New Mexico, United States, 87106
        • Novo Nordisk Investigational Site
    • North Carolina
      • Charlotte, North Carolina, United States, 28203
        • Novo Nordisk Investigational Site
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Probability Sample

Study Population

Previously FVIII treated (more than 150 exposure days) patients with severe and moderately severe haemophilia A with FVIII below or equal to 2%.

Description

Inclusion Criteria:

  • Informed consent obtained before any study-related activities. Study-related activities are any procedure related to recording of data according to the protocol
  • Previously FVIII treated (150 exposure days at the time of first dosing with turoctocog alfa) male patients with the diagnosis of severe and moderately severe haemophilia A (FVIII below or equal to 2%)
  • The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient's treating physician before and independently from the decision to include the patient in this study
  • A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa

Exclusion Criteria:

  • Contraindications for use according to the approved product information text (US Package insert (PI), European Summary of Product Characteristics (SmPC), or corresponding local prescribing information)
  • Treatment with any investigational drug within 30 days prior to enrolment into the study
  • Previous participation in any clinical trial with turoctocog alfa
  • Treatment with other FVIII products after initiation of treatment with turoctocog alfa

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
All patients
Drug: turoctocog alfa
Patients will be treated with commercially available turoctocog alfa as prescribed by the treating physician in clinical daily practice and preferably according to the label for turoctocog alfa in the respective countries.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors
Time Frame: Within approximately 7 years
Within approximately 7 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Number of adverse reactions reported
Time Frame: During approximately 7 years
During approximately 7 years
Number of serious adverse reactions reported
Time Frame: During approximately 7 years
During approximately 7 years
Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the physician according to a predefined four point scale: Excellent, Good, Moderate, or None
Time Frame: Within approximately 7 years
Within approximately 7 years
Haemostatic effect of turoctocog alfa during surgical procedures as assessed by an evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None
Time Frame: Within approximately 7 years
Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for preventive treatment
Time Frame: Within approximately 7 years
Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
Time Frame: Within approximately 7 years
Within approximately 7 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 5, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 15, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 15, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 10, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 10, 2014

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 14, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 27, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 24, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NN7008-3553
  • U1111-1126-0353 (Other Identifier: WHO)
  • ENCEPP/SDPP/5501 (Registry Identifier: EU PAS)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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