NCT00703911|CompletedResults

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

ONE

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

Novo Nordisk A/S ClinicalTrials.gov

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1 other identifier

F7HAEM-3507

Study Type

observational

Target

102

Locations

16 countries

Sites

Timeline

RegisteredJun 2008

StartedMar 2008

CompletionJul 2010

Brief Summary

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

102

participants targeted

Target at P50-P75 for all trials

Timeline

Completed

Started Mar 2008

Typical duration for all trials

Geographic Reach

16 countries

16 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.3 years study duration

Study Start

First participant enrolled

March 1, 2008

Completed

4 months until next milestone

First Submitted

Initial submission to the registry

June 19, 2008

Completed

5 days until next milestone

First Posted

Study publicly available on registry

June 24, 2008

Completed

2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2010

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2010

Completed

1.5 years until next milestone

Results Posted

Study results publicly available

December 19, 2011

Completed

Last Updated

January 11, 2017

Status Verified

November 1, 2016

Enrollment Period

2.3 years

First QC Date

June 19, 2008

Results QC Date

July 29, 2011

Last Update Submit

November 22, 2016

Conditions

Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Outcome Measures

Primary Outcomes (4)

Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
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)
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)
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)
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

Secondary Outcomes (17)

Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
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)
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)
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)
1 hour, 3 hours and 6 hours, respectively, after first injection
Total Number of Injections (All Bleed Episodes)
individual bleed episode
+12 more secondary outcomes

Other Outcomes (4)

Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
within 9 hours after first injection
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
within 9 hours after first injection
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
within 9 hours after first injection
+1 more other outcomes

Study Arms (1)

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

Drug: eptacog alfa (activated)

Interventions

eptacog alfa (activated)DRUG

Treatment of patients experiencing bleeds at the discretion of the physician/caregiver

Also known as: activated recombinant human factor VII

activated recombinant human factor VII

Eligibility Criteria

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

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

You may qualify if:

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)

You may not qualify if:

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

Contact the study team to confirm eligibility.