Observational Patient Diary Study of Treatment Doses for Patients With Haemophilia With Inhibitors to Factors VIII and IX

Dosing Observational Study in Hemophilia Subjects With Inhibitors: A Phase IV Diary Study in Subjects Prescribed NovoSeven® as First Line on Demand Therapy for Acute Bleeding Episodes

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00710619

Acronym

DOSE

Enrollment

Registered

2008-07-04

Start date

2008-06-30

Completion date

2009-07-31

Last updated

2016-11-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors

Brief summary

This study is conducted in the United States of America (USA). The aim of this study is to investigate the at-home-administration of bypassing agents for treatment of bleeding episodes in patients with congenital haemophilia with inhibitors to factors VIII and IX. We are further investigating how bleeding episodes affect the quality of life of the patient and their family or caregivers.

Interventions

DRUGactivated recombinant human factor VII

This is an observation of how patients/caregivers dose bypassing agents at home for haemophilia with inhibitors

DRUGFeiba VH

This is an observation of how patients/caregivers dose bypassing agents at home for hemophilia with inhibitors

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Healthy volunteers

Inclusion criteria

* Male subjects with congenital haemophilia A or B and inhibitors with spontaneous bleeds which require on-demand treatment * Subjects prescribed NovoSeven® as the first line or recommended bypass agent * History of on average at least 4 bleeds of any type over a 3 month period * Subject or caregiver able and willing to complete daily journal for 3 months * Informed consent obtained from all subjects or legal representative

Design outcomes

Primary

Measure	Time frame
Characterisation of dose and dosing intervals for each bleeding episode	after 3-6 months

Secondary

Measure	Time frame
Effect of type of bleed on initial dose, dosing interval and total dose	after 3-6 months
Effect of initial dose and dosing interval on total dose, time to first perceived improvement, time to perceived bleed resolution	after 3-6 months
Effect of time to first dose on dosing interval and total dose	after 3-6 months
Effect of bleeds and drug administration time on planned daily activities	after 3-6 months
Relationship of dose and dosing intervals to reported SAEs	after 3-6 months

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026