Clinical trial NCT00703911: Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2008-06-24
Enrollment: 102
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Time perspective: PROSPECTIVE
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

Brief 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.


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that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)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

Primary outcomes:
measure: Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
timeFrame: within 9 hours of first injection
measure: Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
timeFrame: within 9 hours of first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
timeFrame: within 9 hours of first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
timeFrame: within 9 hours of first injection
Secondary outcomes:
measure: Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
timeFrame: 1 hour, 3 hours and 6 hours, respectively, after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
timeFrame: 1 hour, 3 hours and 6 hours, respectively, after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
timeFrame: 1 hour, 3 hours and 6 hours, respectively, after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
timeFrame: 1 hour, 3 hours and 6 hours, respectively, after first injection
measure: Total Number of Injections (All Bleed Episodes)
timeFrame: individual bleed episode
measure: Total Number of Injections (Spontaneous Bleed Episodes)
timeFrame: individual bleed episode
measure: Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
timeFrame: individual bleed episode
measure: Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
timeFrame: individual bleed episode
measure: Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
timeFrame: duration of bleed episode
measure: Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
timeFrame: duration of bleed episode
measure: Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
timeFrame: duration of bleed episode
measure: Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
timeFrame: duration of bleed episode
measure: Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
timeFrame: duration of bleed episode
measure: Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
timeFrame: duration of bleed episode
measure: Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
timeFrame: duration of bleed episode
measure: Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score
timeFrame: Baseline (week 0) and and registry discontinuation (up to 28 months)
measure: Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score
timeFrame: Baseline (week 0) and and registry discontinuation (up to 28 months)
Other outcomes:
measure: Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
timeFrame: within 9 hours after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
timeFrame: within 9 hours after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
timeFrame: within 9 hours after first injection
measure: Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
timeFrame: within 9 hours after first injection
Locations:
city: Algiers
country: Algeria
facility: Novo Nordisk Investigational Site
city: Vienna
country: Austria
facility: Novo Nordisk Investigational Site
city: Brussels
country: Belgium
facility: Novo Nordisk Investigational Site
city: Prague
country: Czech Republic
facility: Novo Nordisk Investigational Site
city: Paris La défense cedex
country: France
facility: Novo Nordisk Investigational Site
city: Mainz
country: Germany
facility: Novo Nordisk Investigational Site
city: Rome
country: Italy
facility: Novo Nordisk Investigational Site
city: Alphen a/d Rijn
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Warszawa
country: Poland
facility: Novo Nordisk Investigational Site
city: Paco de Arcos
country: Portugal
facility: Novo Nordisk Investigational Site
city: Riyadh
country: Saudi Arabia
facility: Novo Nordisk Investigational Site
city: Sandton
country: South Africa
facility: Novo Nordisk Investigational Site
city: Malmö
country: Sweden
facility: Novo Nordisk Investigational Site
city: Istanbul
country: Turkey
facility: Novo Nordisk Investigational Site
city: Crawley
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Caracas
country: Venezuela
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: