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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 novembre 2016 aggiornato da: 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.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Tipo di studio

Osservativo

Iscrizione (Effettivo)

102

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Algeria
      • Algiers, Algeria, 16035
        • Novo Nordisk Investigational Site
  • Arabia Saudita
      • Riyadh, Arabia Saudita, 3542
        • Novo Nordisk Investigational Site
  • Austria
      • Vienna, Austria, A-1010
        • Novo Nordisk Investigational Site
  • Belgio
      • Brussels, Belgio, 1070
        • Novo Nordisk Investigational Site
  • Francia
      • Paris La défense cedex, Francia, 92932
        • Novo Nordisk Investigational Site
  • Germania
      • Mainz, Germania, 55127
        • Novo Nordisk Investigational Site
  • Italia
      • Rome, Italia, 00144
        • Novo Nordisk Investigational Site
  • Olanda
      • Alphen a/d Rijn, Olanda
        • Novo Nordisk Investigational Site
  • Polonia
      • Warszawa, Polonia, PL-02-274
        • Novo Nordisk Investigational Site
  • Portogallo
      • Paco de Arcos, Portogallo, 2780-730
        • Novo Nordisk Investigational Site
  • Regno Unito
      • Crawley, Regno Unito, RH11 9RT
        • Novo Nordisk Investigational Site
  • Repubblica Ceca
      • Prague, Repubblica Ceca, 16000
        • Novo Nordisk Investigational Site
  • Sud Africa
      • Sandton, Sud Africa, 2146
        • Novo Nordisk Investigational Site
  • Svezia
      • Malmö, Svezia, 202 15
        • Novo Nordisk Investigational Site
  • Tacchino
      • Istanbul, Tacchino, 34335
        • Novo Nordisk Investigational Site
  • Venezuela
      • Caracas, Venezuela
        • Novo Nordisk Investigational Site

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Sessi ammissibili allo studio

Maschio

Metodo di campionamento

Campione non probabilistico

Popolazione di studio

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

Descrizione

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

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

Coorti e interventi

Gruppo / Coorte
Intervento / Trattamento
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
Droga: eptacog alfa (activated)
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
Altri nomi:
  • fattore umano ricombinante attivato VII

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: individual bleed episode
The median number of injections required to treat individual bleed episodes.
individual bleed episode
Total Number of Injections (Spontaneous Bleed Episodes)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)

Altre misure di risultato

Misura del risultato
Misura Descrizione
Lasso di tempo
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Novo Nordisk A/S

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Collegamenti utili

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio

1 marzo 2008

Completamento primario (Effettivo)

1 luglio 2010

Completamento dello studio (Effettivo)

1 luglio 2010

Date di iscrizione allo studio

Primo inviato

19 giugno 2008

Primo inviato che soddisfa i criteri di controllo qualità

23 giugno 2008

Primo Inserito (Stima)

24 giugno 2008

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Stima)

11 gennaio 2017

Ultimo aggiornamento inviato che soddisfa i criteri QC

22 novembre 2016

Ultimo verificato

1 novembre 2016

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • F7HAEM-3507

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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