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

Purpose

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	Intervention
Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors	Drug: activated recombinant human factor VII Drug: Feiba VH


Study Type:	Observational
Study Design:	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

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia

MedlinePlus related topics: Bleeding Hemophilia

Genetic and Rare Diseases Information Center resources: Hemophilia Hemophilia A Hemophilia B

U.S. FDA Resources

Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:

Characterisation of dose and dosing intervals for each bleeding episode [ Time Frame: after 3-6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Effect of type of bleed on initial dose, dosing interval and total dose [ Time Frame: after 3-6 months ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
Effect of time to first dose on dosing interval and total dose [ Time Frame: after 3-6 months ] [ Designated as safety issue: No ]
Effect of bleeds and drug administration time on planned daily activities [ Time Frame: after 3-6 months ] [ Designated as safety issue: No ]
Relationship of dose and dosing intervals to reported SAEs [ Time Frame: after 3-6 months ] [ Designated as safety issue: No ]


Enrollment:	52
Study Start Date:	June 2008
Study Completion Date:	July 2009
Primary Completion Date:	July 2009 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
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

Eligibility


Ages Eligible for Study:	Child, Adult, Senior
Genders 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00710619

Locations


United States, New Jersey
Novo Nordisk Clinical Trial Call Center
Princeton, New Jersey, United States, 08540

Sponsors and Collaborators

Novo Nordisk A/S

Investigators


Study Director:	David L. Cooper, MD	Novo Nordisk A/S

More Information

Additional Information:

Clinical Trials at Novo Nordisk This link exits the ClinicalTrials.gov site

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
Study First Received:	July 3, 2008
Last Updated:	August 30, 2012
Health Authority:	United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on September 26, 2016

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