PROPACT: Retrospective Prophylaxis Patient Case Collection

Congenital Bleeding Disorder Clinical Trial

— PROPACT  

Official title:

Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00882778
• Other study ID #: F7HAEM-3695
• Status: Completed
• Phase: N/A
• First received: April 14, 2009
• Last updated: August 6, 2014
• Start date: April 2009
• Est. completion date: May 2010

Study information

• Verified date: August 2014
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia MedicaCanada: Public Health Agency of CanadaCroatia: Ministry of Health and Social CareCzech Republic: State Institute for Drug ControlFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: BfArM and Paul-Ehrlich Institute (PEI)Ireland: Irish Medicines BoardItaly: AIFASlovakia: State Institute for Drug ControlSpain: AGEMEDSweden: Medical Products AgencySwitzerland: Federal Office of Public HealthUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyUnited States: Food and Drug Administration
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 86
• Est. completion date: May 2010
• Est. primary completion date: May 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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

Study Design

Observational Model: Case-Only, Time Perspective: Retrospective

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
Retrospective data collection of the use of activated recombinant human factor VII as prophylaxis in haemophilia patients with inhibitors

Locations

Country	Name	City	State
United States	Novo Nordisk Clinical Trial Call Center	Princeton	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Argentina,  Canada,  Croatia,  Czech Republic,  France,  Germany,  Ireland,  Italy,  Slovakia,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change in Total Bleed Episodes Per Month - Bleeding Population	Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population	Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Primary	Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric	Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent	Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult	Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric	Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent	Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult	Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	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.	No
Secondary	Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population	Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (defined as 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.	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.	No
Secondary	Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population	Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (= 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.	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.	No
Secondary	Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period.	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.	No
Secondary	Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.	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.	No
Secondary	Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.	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.	No
Secondary	Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	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.	No
Secondary	Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	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.	No
Secondary	Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	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.	No
Secondary	Physician Reported Outcome Assessment in Prophylaxis in Number of Patients	Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine	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.	No
Secondary	Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients	Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine	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.	No

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