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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. november 2016 oppdatert av: 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.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Studietype

Observasjonsmessig

Registrering (Faktiske)

102

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Algerie
      • Algiers, Algerie, 16035
        • Novo Nordisk Investigational Site
  • Belgia
      • Brussels, Belgia, 1070
        • Novo Nordisk Investigational Site
  • Frankrike
      • Paris La défense cedex, Frankrike, 92932
        • Novo Nordisk Investigational Site
  • Italia
      • Rome, Italia, 00144
        • Novo Nordisk Investigational Site
  • Nederland
      • Alphen a/d Rijn, Nederland
        • Novo Nordisk Investigational Site
  • Polen
      • Warszawa, Polen, PL-02-274
        • Novo Nordisk Investigational Site
  • Portugal
      • Paco de Arcos, Portugal, 2780-730
        • Novo Nordisk Investigational Site
  • Saudi-Arabia
      • Riyadh, Saudi-Arabia, 3542
        • Novo Nordisk Investigational Site
  • Storbritannia
      • Crawley, Storbritannia, RH11 9RT
        • Novo Nordisk Investigational Site
  • Sverige
      • Malmö, Sverige, 202 15
        • Novo Nordisk Investigational Site
  • Sør-Afrika
      • Sandton, Sør-Afrika, 2146
        • Novo Nordisk Investigational Site
  • Tsjekkisk Republikk
      • Prague, Tsjekkisk Republikk, 16000
        • Novo Nordisk Investigational Site
  • Tyrkia
      • Istanbul, Tyrkia, 34335
        • Novo Nordisk Investigational Site
  • Tyskland
      • Mainz, Tyskland, 55127
        • Novo Nordisk Investigational Site
  • Venezuela
      • Caracas, Venezuela
        • Novo Nordisk Investigational Site
  • Østerrike
      • Vienna, Østerrike, A-1010
        • Novo Nordisk Investigational Site

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Mann

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

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

Beskrivelse

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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Kohorter og intervensjoner

Gruppe / Kohort
Intervensjon / Behandling
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
Legemiddel: eptacog alfa (activated)
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
Andre navn:
  • aktivert rekombinant human faktor VII

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: individual bleed episode
The median number of injections required to treat individual bleed episodes.
individual bleed episode
Total Number of Injections (Spontaneous Bleed Episodes)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Novo Nordisk A/S

Publikasjoner og nyttige lenker

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Hjelpsomme linker

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. mars 2008

Primær fullføring (Faktiske)

1. juli 2010

Studiet fullført (Faktiske)

1. juli 2010

Datoer for studieregistrering

Først innsendt

19. juni 2008

Først innsendt som oppfylte QC-kriteriene

23. juni 2008

Først lagt ut (Anslag)

24. juni 2008

Oppdateringer av studieposter

Sist oppdatering lagt ut (Anslag)

11. januar 2017

Siste oppdatering sendt inn som oppfylte QC-kriteriene

22. november 2016

Sist bekreftet

1. november 2016

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • F7HAEM-3507

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