PROPACT: Retrospective Prophylaxis Patient Case Collection

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00882778
First received: April 14, 2009
Last updated: August 6, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention
Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Drug: activated recombinant human factor VII

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Percent Change in Total Bleed Episodes Per Month - Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period

  • Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period

  • Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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

  • Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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).

  • Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population [ Time Frame: 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. ] [ Designated as safety issue: No ]
    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.

  • Physician Reported Outcome Assessment in Prophylaxis in Number of Patients [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine

  • Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients [ Time Frame: 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. ] [ Designated as safety issue: No ]
    Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine


Enrollment: 86
Study Start Date: April 2009
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
activated recombinant human factor VII
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Haemophilia patients in specialist hospital clinic or private clinic settings

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00882778

Locations
United States, New Jersey
Novo Nordisk Clinical Trial Call Center
Princeton, New Jersey, United States, 08540
Argentina
Prov. de Buenos Aires, Argentina, B1636DSU
Canada
Mississauga, Canada, L4W 4XI
Croatia
Zagreb, Croatia, 10 000
Czech Republic
Prague, Czech Republic, 16000
France
Paris La défense cedex, France, 92932
Germany
Mainz, Germany, 55127
Ireland
Dublin 2, Ireland
Italy
Rome, Italy, 00144
Slovakia
Bratislava, Slovakia, 811 05
Spain
Madrid, Spain, 28033
Sweden
Malmö, Sweden, 202 15
Switzerland
Zurich, Switzerland, CH-8050
United Kingdom
Crawley, United Kingdom, RH11 9RT
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Pedro Pina, MD Novo Nordisk Health Care AG
  More Information

Additional Information:
No publications provided

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00882778     History of Changes
Other Study ID Numbers: F7HAEM-3695
Study First Received: April 14, 2009
Results First Received: May 11, 2011
Last Updated: August 6, 2014
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Canada: Public Health Agency of Canada
Croatia: Ministry of Health and Social Care
Czech Republic: State Institute for Drug Control
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: BfArM and Paul-Ehrlich Institute (PEI)
Ireland: Irish Medicines Board
Italy: AIFA
Slovakia: State Institute for Drug Control
Spain: AGEMED
Sweden: Medical Products Agency
Switzerland: Federal Office of Public Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hemostatic Disorders
Hemorrhagic Disorders
Hemophilia B
Hemophilia A
Hemorrhage
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Pathologic Processes

ClinicalTrials.gov processed this record on August 27, 2014