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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 uppdaterad 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.

Studieöversikt

Status

Avslutad

Betingelser

Intervention / Behandling

Studietyp

Observationell

Inskrivning (Faktisk)

102

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • Algeriet
      • Algiers, Algeriet, 16035
        • Novo Nordisk Investigational Site
  • Belgien
      • Brussels, Belgien, 1070
        • Novo Nordisk Investigational Site
  • Frankrike
      • Paris La défense cedex, Frankrike, 92932
        • Novo Nordisk Investigational Site
  • Italien
      • Rome, Italien, 00144
        • Novo Nordisk Investigational Site
  • Kalkon
      • Istanbul, Kalkon, 34335
        • Novo Nordisk Investigational Site
  • Nederländerna
      • Alphen a/d Rijn, Nederländerna
        • 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
  • Saudiarabien
      • Riyadh, Saudiarabien, 3542
        • Novo Nordisk Investigational Site
  • Storbritannien
      • Crawley, Storbritannien, RH11 9RT
        • Novo Nordisk Investigational Site
  • Sverige
      • Malmö, Sverige, 202 15
        • Novo Nordisk Investigational Site
  • Sydafrika
      • Sandton, Sydafrika, 2146
        • Novo Nordisk Investigational Site
  • Tjeckien
      • Prague, Tjeckien, 16000
        • Novo Nordisk Investigational Site
  • Tyskland
      • Mainz, Tyskland, 55127
        • Novo Nordisk Investigational Site
  • Venezuela
      • Caracas, Venezuela
        • Novo Nordisk Investigational Site
  • Österrike
      • Vienna, Österrike, A-1010
        • Novo Nordisk Investigational Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Barn
  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Manlig

Testmetod

Icke-sannolikhetsprov

Studera befolkning

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

Beskrivning

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

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Kohorter och interventioner

Grupp / Kohort
Intervention / 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
Läkemedel: eptacog alfa (activated)
Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
Andra namn:
  • aktiverad rekombinant human faktor VII

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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

Sekundära resultatmått

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

Andra resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Novo Nordisk A/S

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Användbara länkar

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart

1 mars 2008

Primärt slutförande (Faktisk)

1 juli 2010

Avslutad studie (Faktisk)

1 juli 2010

Studieregistreringsdatum

Först inskickad

19 juni 2008

Först inskickad som uppfyllde QC-kriterierna

23 juni 2008

Första postat (Uppskatta)

24 juni 2008

Uppdateringar av studier

Senaste uppdatering publicerad (Uppskatta)

11 januari 2017

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

22 november 2016

Senast verifierad

1 november 2016

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • F7HAEM-3507

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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