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

22. listopadu 2016 aktualizováno: Novo Nordisk A/S

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

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.

Přehled studie

Postavení

Dokončeno

Podmínky

Intervence / Léčba

Typ studie

Pozorovací

Zápis (Aktuální)

102

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

  • Alžírsko
      • Algiers, Alžírsko, 16035
        • Novo Nordisk Investigational Site
  • Belgie
      • Brussels, Belgie, 1070
        • Novo Nordisk Investigational Site
  • Francie
      • Paris La défense cedex, Francie, 92932
        • Novo Nordisk Investigational Site
  • Holandsko
      • Alphen a/d Rijn, Holandsko
        • Novo Nordisk Investigational Site
  • Itálie
      • Rome, Itálie, 00144
        • Novo Nordisk Investigational Site
  • Jižní Afrika
      • Sandton, Jižní Afrika, 2146
        • Novo Nordisk Investigational Site
  • Krocan
      • Istanbul, Krocan, 34335
        • Novo Nordisk Investigational Site
  • Německo
      • Mainz, Německo, 55127
        • Novo Nordisk Investigational Site
  • Polsko
      • Warszawa, Polsko, PL-02-274
        • Novo Nordisk Investigational Site
  • Portugalsko
      • Paco de Arcos, Portugalsko, 2780-730
        • Novo Nordisk Investigational Site
  • Rakousko
      • Vienna, Rakousko, A-1010
        • Novo Nordisk Investigational Site
  • Saudská arábie
      • Riyadh, Saudská arábie, 3542
        • Novo Nordisk Investigational Site
  • Spojené království
      • Crawley, Spojené království, RH11 9RT
        • Novo Nordisk Investigational Site
  • Venezuela
      • Caracas, Venezuela
        • Novo Nordisk Investigational Site
  • Česká republika
      • Prague, Česká republika, 16000
        • Novo Nordisk Investigational Site
  • Švédsko
      • Malmö, Švédsko, 202 15
        • Novo Nordisk Investigational Site

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

  • Dítě
  • Dospělý
  • Starší dospělý

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Mužský

Metoda odběru vzorků

Vzorek nepravděpodobnosti

Studijní populace

Patients with haemophilia A or B with inhibitors, using activated recombinant human factor VII as on-demand treatment

Popis

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

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

Kohorty a intervence

Skupina / kohorta
Intervence / Léčba
activated recombinant human factor VII
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
Lék: eptacog alfa (activated)
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
Ostatní jména:
  • aktivovaný rekombinantní lidský faktor VII

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Časové okno: within 9 hours of first injection
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
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
Časové okno: within 9 hours of first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
Časové okno: within 9 hours of first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
Časové okno: within 9 hours of first injection
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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Časové okno: 1 hour, 3 hours and 6 hours, respectively, after first injection
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
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Časové okno: 1 hour, 3 hours and 6 hours, respectively, after first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Časové okno: 1 hour, 3 hours and 6 hours, respectively, after first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Časové okno: 1 hour, 3 hours and 6 hours, respectively, after first injection
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
Total Number of Injections (All Bleed Episodes)
Časové okno: individual bleed episode
The median number of injections required to treat individual bleed episodes.
individual bleed episode
Total Number of Injections (Spontaneous Bleed Episodes)
Časové okno: individual bleed episode
The median number of injections required to treat individual bleed episodes.
individual bleed episode
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
Časové okno: individual bleed episode
The median total cumulative dose required to treat individual bleed episodes.
individual bleed episode
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
Časové okno: individual bleed episode
The median total cumulative dose required to treat individual bleed episodes.
individual bleed episode
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
Časové okno: duration of bleed episode
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
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
Časové okno: duration of bleed episode
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
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
Časové okno: duration of bleed episode
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
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
Časové okno: duration of bleed episode
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
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
Časové okno: duration of bleed episode
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
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Časové okno: duration of bleed episode
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
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Časové okno: duration of bleed episode
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
Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score
Časové okno: Baseline (week 0) and and registry discontinuation (up to 28 months)
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)
Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score
Časové okno: Baseline (week 0) and and registry discontinuation (up to 28 months)
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)

Další výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Časové okno: within 9 hours after first injection
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
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Časové okno: within 9 hours after first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Časové okno: within 9 hours after first injection
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
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Časové okno: within 9 hours after first injection
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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Novo Nordisk A/S

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Užitečné odkazy

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia

1. března 2008

Primární dokončení (Aktuální)

1. července 2010

Dokončení studie (Aktuální)

1. července 2010

Termíny zápisu do studia

První předloženo

19. června 2008

První předloženo, které splnilo kritéria kontroly kvality

23. června 2008

První zveřejněno (Odhad)

24. června 2008

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Odhad)

11. ledna 2017

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

22. listopadu 2016

Naposledy ověřeno

1. listopadu 2016

Více informací

Termíny související s touto studií

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • F7HAEM-3507

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

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Klinické studie na eptacog alfa (activated)

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Sponzoři a spolupracovníci

Zdravotní podmínky

Drogové intervence

CROs by country

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Podmínky

Vzácné nemoci

Drogové intervence

Doplňky stravy

Sponzor / Spolupracovníci

Místa

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