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Completed

NCT Number: NCT00703911

Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B 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.

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Key information

Conditions

Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Sex eligibility

Male

Study type

Observational

Primary location

Novo Nordisk Investigational Site, Algiers, Algeria

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Who can participate

Healthy volunteers accepted: No

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

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that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)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

Treatment and study plan

eptacog alfa (activated)

Drug

Treatment of patients experiencing bleeds at the discretion of the physician/caregiver

Other names: activated recombinant human factor VII

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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 outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. Total Number of Injections (All Bleed Episodes)

    Time frame: individual bleed episode

    The median number of injections required to treat individual bleed episodes.

  6. Total Number of Injections (Spontaneous Bleed Episodes)

    Time frame: individual bleed episode

    The median number of injections required to treat individual bleed episodes.

  7. 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.

  8. 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.

  9. 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.

  10. 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.

  11. 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.

  12. 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.

  13. 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

  14. 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

  15. 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.

  16. 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.

  17. 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 outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study 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

Acronym: ONE

Important dates

Study start
2008
Primary completion
2010
Study completion
2010
First posted
Jun 24, 2008
Registry last updated
Jan 11, 2017

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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