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

Congenital Bleeding Disorder Clinical Trial

— ONE  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00703911
• Other study ID #: F7HAEM-3507
• Status: Completed
• Phase: N/A
• First received: June 19, 2008
• Last updated: August 6, 2014
• Start date: March 2008
• Est. completion date: July 2010

Study information

• Verified date: August 2014
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Algeria: Ministry of HealthAustria: The Austrian Agency for Health and Food Safety (AGES)Belgium: Federal Agency for Medicines and Healthcare ProductsCzech Republic: State Institute for Drug ControlFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Federal Institute for Drugs and Medicinal Devices (BfarM)Italy: AIFA, National Medicines AgencyNetherlands: The Central Committee on Research Involving Human Subjects (CCMO)Poland: The Office for Reg. of Medicinal Products, Medical Devices and Biocidal Products - Central Register of Clinical TrialsPortugal: INFARMEDSaudi Arabia: Ministry of HealthSouth Africa: Medicines Control CouncilSweden: Medical Products AgencyTurkey: Ministry of Health Drug and Pharmaceutical DepartmentUnited Kingdom: Medicines and Healthcare Regulatory Authority (MHRA)Venezuela: Ministry of Health
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 102
• Est. completion date: July 2010
• Est. primary completion date: July 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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

Study Design

Time Perspective: Prospective

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Haemophilia A With Inhibitors
• Haemophilia B With Inhibitors
• Hemophilia A
• Hemophilia B
• Hemostatic Disorders

Intervention

Drug:
• activated recombinant human factor VII
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

Algeria,  Austria,  Belgium,  Czech Republic,  France,  Germany,  Italy,  Netherlands,  Poland,  Portugal,  Saudi Arabia,  South Africa,  Sweden,  Turkey,  United Kingdom,  Venezuela, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)	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.	within 9 hours after first injection	No
Other	Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)	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.	within 9 hours after first injection	No
Other	Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)	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.	within 9 hours after first injection	No
Other	Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)	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.	within 9 hours after first injection	No
Primary	Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)	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.	within 9 hours of first injection	No
Primary	Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)	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.	within 9 hours of first injection	No
Primary	Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)	The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.	within 9 hours of first injection	No
Primary	Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)	The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.	within 9 hours of first injection	No
Secondary	Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)	Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.	1 hour, 3 hours and 6 hours, respectively, after first injection	No
Secondary	Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)	Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.	1 hour, 3 hours and 6 hours, respectively, after first injection	No
Secondary	Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)	Effective pain relief at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.	1 hour, 3 hours and 6 hours, respectively, after first injection	No
Secondary	Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)	Effective pain relief at 3 different time points for spontaneous bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.	1 hour, 3 hours and 6 hours, respectively, after first injection	No
Secondary	Total Number of Injections (All Bleed Episodes)	The median number of injections required to treat individual bleed episodes.	individual bleed episode	No
Secondary	Total Number of Injections (Spontaneous Bleed Episodes)	The median number of injections required to treat individual bleed episodes.	individual bleed episode	No
Secondary	Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)	The median total cumulative dose required to treat individual bleed episodes.	individual bleed episode	No
Secondary	Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)	The median total cumulative dose required to treat individual bleed episodes.	individual bleed episode	No
Secondary	Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)	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.	duration of bleed episode	No
Secondary	Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)	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.	duration of bleed episode	No
Secondary	Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)	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.	duration of bleed episode	No
Secondary	Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)	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.	duration of bleed episode	No
Secondary	Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)	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	duration of bleed episode	No
Secondary	Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)	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	duration of bleed episode	No
Secondary	Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)	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.	duration of bleed episode	No
Secondary	Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score	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.	Baseline (week 0) and and registry discontinuation (up to 28 months)	No
Secondary	Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score	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.	Baseline (week 0) and and registry discontinuation (up to 28 months)	No

External Links

•   Clinical Trials at Novo Nordisk