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: December 3, 2012
Last verified: December 2012

June 19, 2008
December 3, 2012
March 2008
July 2010   (final data collection date for primary outcome measure)
  • Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes) [ Time Frame: within 9 hours of first injection ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
  • Proportion of bleed treatments resulting in effective pain relief [ Time Frame: 9 hours after first injection, for each bleeding episode ] [ Designated as safety issue: No ]
  • Proportion of bleed treatments resulting in effective haemostasis [ Time Frame: 9 hours after first injection, for each bleeding episode ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00703911 on ClinicalTrials.gov Archive Site
  • 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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    The median number of injections required to treat individual bleed episodes.
  • Total Number of Injections (Spontaneous Bleed Episodes) [ Time Frame: individual bleed episode ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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.
  • Treatment satisfaction and convenience [ Time Frame: after each bleeding episode ] [ Designated as safety issue: No ]
  • Haemostasis/pain relief [ Time Frame: 1 hour, 3 hours and 6 hours after start of each bleed ] [ Designated as safety issue: No ]
  • Adverse events reported as potentially related to NovoSeven [ Time Frame: up to 28 days after discontinuation from the study ] [ Designated as safety issue: Yes ]
  • Total number of injections/doses [ Time Frame: 48 hours after start of each bleed ] [ Designated as safety issue: No ]
  • Quality of Life questionnaire (voluntary) [ Time Frame: at study discontinuation ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.
Not Provided
 
Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors
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

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.

Not Provided
Observational
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample

Patients with haemophilia A or B with inhibitors, using activated recombinant human factor VII as on-demand treatment

  • Congenital Bleeding Disorder
  • Haemophilia A With Inhibitors
  • Haemophilia B With Inhibitors
Drug: activated recombinant human factor VII
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
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
Intervention: Drug: activated recombinant human factor VII
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
102
July 2010
July 2010   (final data collection date for primary outcome measure)

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
Male
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
Algeria,   Austria,   Belgium,   Czech Republic,   France,   Germany,   Italy,   Netherlands,   Poland,   Portugal,   Saudi Arabia,   South Africa,   Sweden,   Turkey,   United Kingdom,   Venezuela
 
NCT00703911
F7HAEM-3507
No
Novo Nordisk A/S
Novo Nordisk A/S
Not Provided
Study Director: Paulo André Palhares de Miranda Novo Nordisk Health Care AG
Novo Nordisk A/S
December 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP