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

Study Description

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.

Condition or disease	Intervention/treatment
Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors	Drug: activated recombinant human factor VII; Drug: Feiba VH

Study Design

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Study Type :	Observational
Actual Enrollment :	52 participants
Observational Model:	Case-Only
Time Perspective:	Prospective
Official Title:	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
Study Start Date :	June 2008
Actual Primary Completion Date :	July 2009
Actual Study Completion Date :	July 2009

Groups and Cohorts

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Group/Cohort	Intervention/treatment
A	Drug: activated recombinant human factor VII; This is an observation of how patients/caregivers dose bypassing agents at home for haemophilia with inhibitors Drug: Feiba VH; This is an observation of how patients/caregivers dose bypassing agents at home for hemophilia with inhibitors

Outcome Measures

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. Characterisation of dose and dosing intervals for each bleeding episode [ Time Frame: after 3-6 months ]

Secondary Outcome Measures :

1. Effect of type of bleed on initial dose, dosing interval and total dose [ Time Frame: after 3-6 months ]
2. Effect of initial dose and dosing interval on total dose, time to first perceived improvement, time to perceived bleed resolution [ Time Frame: after 3-6 months ]
3. Effect of time to first dose on dosing interval and total dose [ Time Frame: after 3-6 months ]
4. Effect of bleeds and drug administration time on planned daily activities [ Time Frame: after 3-6 months ]
5. Relationship of dose and dosing intervals to reported SAEs [ Time Frame: after 3-6 months ]

Eligibility Criteria

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	Child, Adult, Senior
Sexes Eligible for Study:	Male
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Male subjects with congenital haemophilia A or B with inhibitors with spontaneous bleeds requiring on-demand treatment

Criteria

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

Contacts and Locations

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

United States, New Jersey
Novo Nordisk Investigational Site
Plainsboro, New Jersey, United States, 08536

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

Study Director:	Global Clinical Registry (GCR, 1452)	Novo Nordisk A/S

More Information

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:

Clinical Trials at Novo Nordisk 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Gruppo RA, Kessler CM, Neufeld EJ, Cooper DL. Assessment of individual dose utilization vs. physician prescribing recommendations for recombinant activated factor VII (rFVIIa) in paediatric and adult patients with congenital haemophilia and alloantibody inhibitors (CHwI): the Dosing Observational Study in Hemophilia (DOSE). Haemophilia. 2013 Jul;19(4):524-32. doi: 10.1111/hae.12113. Epub 2013 Apr 1.

Responsible Party:	Novo Nordisk A/S
ClinicalTrials.gov Identifier:	NCT00710619 History of Changes
Other Study ID Numbers:	F7HAEM-1965
First Posted:	July 4, 2008
Last Update Posted:	November 17, 2016
Last Verified:	November 2016

Additional relevant MeSH terms:

Hemophilia A; Hemophilia B; Hemorrhage; Blood Coagulation Disorders; Hemostatic Disorders; Pathologic Processes; Blood Coagulation Disorders, Inherited	Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Vascular Diseases; Cardiovascular Diseases