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

NCT00710619 | Completed

• 1 other identifier: F7HAEM-1965
• Study Type: observational
• Target: 52
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Characterisation of dose and dosing intervals for each bleeding episode

  after 3-6 months

Secondary Outcomes (5)

• 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

Study Arms (1)

A

Drug: activated recombinant human factor VII; Drug: Feiba VH

Interventions

activated recombinant human factor VII DRUG

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

A

Feiba VH DRUG

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

A

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (1)

Novo Nordisk Investigational Site

Plainsboro, New Jersey, 08536, United States

Location

Related Publications (1)

• 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.

  PMID: 23551918 DERIVED

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Interventions

anti-inhibitor coagulant complex

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2008
• First Posted: July 4, 2008
• Study Start: June 1, 2008
• Primary Completion: July 1, 2009
• Study Completion: July 1, 2009
• Last Updated: November 17, 2016

Record last verified: 2016-11

Locations

US (1)