Clinical trial NCT00882778: PROPACT: Retrospective Prophylaxis Patient Case Collection
Official title: Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia 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: 2009-04-16
Enrollment: 86
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Haemophilia patients in specialist hospital clinic or private clinic settings

Brief summary: This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.


Eligibility criteria: Inclusion Criteria:

* Haemophilia A or B with inhibitors
* Prescribed use of activated recombinant human factor VII for any type of prophylaxis with a duration of at least 30 days

Exclusion Criteria:

* Prophylaxis prescribed post-surgery
* One or more coagulation disorders in addition to haemophilia

Primary outcomes:
measure: Percent Change in Total Bleed Episodes Per Month - Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Secondary outcomes:
measure: Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
timeFrame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
measure: Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
timeFrame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Other outcomes:
Locations:
city: Princeton
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
city: Prov. de Buenos Aires
country: Argentina
facility: Novo Nordisk Investigational Site
city: Mississauga
country: Canada
facility: Novo Nordisk Investigational Site
city: Zagreb
country: Croatia
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: Dublin 2
country: Ireland
facility: Novo Nordisk Investigational Site
city: Rome
country: Italy
facility: Novo Nordisk Investigational Site
city: Bratislava
country: Slovakia
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Malmö
country: Sweden
facility: Novo Nordisk Investigational Site
city: Zurich
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Crawley
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: