This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00703911
First received: June 19, 2008
Last updated: November 22, 2016
Last verified: November 2016
  Purpose
This study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.

Condition Intervention
Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors Drug: eptacog alfa (activated)

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: A Prospective Observational Registry on the Use of NovoSeven® (Activated Recombinant Human Factor VIIa) for on Demand Treatment of Mild to Moderate Bleeds in Haemophilia A and B Patients With Inhibitors

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes) [ Time Frame: within 9 hours of first injection ]
    The percentage of bleed treatments successfully resulting in bleed resolution. Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.

  • Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes) [ Time Frame: within 9 hours of first injection ]
    The percentage of bleed treatments successfully resulting in bleed resolution. Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes) [ Time Frame: within 9 hours of first injection ]
    The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes) [ Time Frame: within 9 hours of first injection ]
    The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.


Secondary Outcome Measures:
  • Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes) [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]
    Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

  • Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes) [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]
    Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes) [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]
    Effective pain relief at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes) [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]
    Effective pain relief at 3 different time points for spontaneous bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

  • Total Number of Injections (All Bleed Episodes) [ Time Frame: individual bleed episode ]
    The median number of injections required to treat individual bleed episodes.

  • Total Number of Injections (Spontaneous Bleed Episodes) [ Time Frame: individual bleed episode ]
    The median number of injections required to treat individual bleed episodes.

  • Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes) [ Time Frame: individual bleed episode ]
    The median total cumulative dose required to treat individual bleed episodes.

  • Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes) [ Time Frame: individual bleed episode ]
    The median total cumulative dose required to treat individual bleed episodes.

  • Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely satisfied to extremely dissatisfied. The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.

  • Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely satisfied to extremely dissatisfied. The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.

  • Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely difficult to extremely easy. The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.

  • Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely difficult to extremely easy. The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.

  • Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable

  • Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable

  • Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes) [ Time Frame: duration of bleed episode ]
    Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable.

  • Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score [ Time Frame: Baseline (week 0) and and registry discontinuation (up to 28 months) ]
    The Haemo-QoL is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia. Scores are reported on a 0 to 100 scale—higher scores indicate more impairment.

  • Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score [ Time Frame: Baseline (week 0) and and registry discontinuation (up to 28 months) ]
    The adult Haemo-QoL-A is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia. Scores are reported on a 0 to 100 scale—higher scores indicate more impairment.


Other Outcome Measures:
  • Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes) [ Time Frame: within 9 hours after first injection ]
    Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.

  • Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes) [ Time Frame: within 9 hours after first injection ]
    Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes) [ Time Frame: within 9 hours after first injection ]
    Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.

  • Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes) [ Time Frame: within 9 hours after first injection ]
    Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.


Enrollment: 102
Study Start Date: March 2008
Study Completion Date: July 2010
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
activated recombinant human factor VII
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
Drug: eptacog alfa (activated)
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
Other Name: activated recombinant human factor VII

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with haemophilia A or B with inhibitors, using activated recombinant human factor VII as on-demand treatment
Criteria

Inclusion Criteria:

  • Diagnosed with haemophilia A or B with inhibitors
  • Experience mild to moderate spontaneous bleeds which require on-demand treatment and who are currently prescribed activated recombinant human factor VII
  • Be able and willing to provide informed consent (or proxy consent by caregiver, if applicable), as required by local research ethics committee, governmental or regulatory authorities
  • Be willing to provide information on at least one alternate contact person in the event that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)

Exclusion Criteria:

  • Known hypersensitivity to the active substance or the excipients in the formulation of activated recombinant human factor VII, or to mouse, hamster or bovine protein
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00703911

Locations
Algeria
Novo Nordisk Investigational Site
Algiers, Algeria, 16035
Austria
Novo Nordisk Investigational Site
Vienna, Austria, A-1010
Belgium
Novo Nordisk Investigational Site
Brussels, Belgium, 1070
Czech Republic
Novo Nordisk Investigational Site
Prague, Czech Republic, 16000
France
Novo Nordisk Investigational Site
Paris La défense cedex, France, 92932
Germany
Novo Nordisk Investigational Site
Mainz, Germany, 55127
Italy
Novo Nordisk Investigational Site
Rome, Italy, 00144
Netherlands
Novo Nordisk Investigational Site
Alphen a/d Rijn, Netherlands
Poland
Novo Nordisk Investigational Site
Warszawa, Poland, PL-02-274
Portugal
Novo Nordisk Investigational Site
Paco de Arcos, Portugal, 2780-730
Saudi Arabia
Novo Nordisk Investigational Site
Riyadh, Saudi Arabia, 3542
South Africa
Novo Nordisk Investigational Site
Sandton, South Africa, 2146
Sweden
Novo Nordisk Investigational Site
Malmö, Sweden, 202 15
Turkey
Novo Nordisk Investigational Site
Istanbul, Turkey, 34335
United Kingdom
Novo Nordisk Investigational Site
Crawley, United Kingdom, RH11 9RT
Venezuela
Novo Nordisk Investigational Site
Caracas, Venezuela
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00703911     History of Changes
Other Study ID Numbers: F7HAEM-3507
Study First Received: June 19, 2008
Results First Received: July 29, 2011
Last Updated: November 22, 2016

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases
Factor VIII
Coagulants

ClinicalTrials.gov processed this record on September 21, 2017