Congenital Bleeding DisorderDOSENCT00710619
Observational Patient Diary Study of Treatment Doses for Patients With Haemophilia With Inhibitors to Factors VIII and IX
An observational study of Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- What is being tested?
- activated recombinant human factor VII and Feiba VH
- How many people
- 52 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2008to Jul 2009
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male subjects with congenital haemophilia A or B with inhibitors with spontaneous bleeds requiring on-demand treatment
Who may be able to join
- 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
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Novo Nordisk Investigational Site
Plainsboro, New Jersey, 08536
About this study
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
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
- Being tested
- activated recombinant human factor VII, Feiba VH
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
activated recombinant human factor VIIDrug
This is an observation of how patients/caregivers dose bypassing agents at home for haemophilia with inhibitors
Feiba VHDrug
This is an observation of how patients/caregivers dose bypassing agents at home for hemophilia with inhibitors
| Study group | Role | What they receive |
|---|---|---|
| A | Not described Receives: Drug: activated recombinant human factor VII; Drug: Feiba VH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterisation of dose and dosing intervals for each bleeding episode
Measured: after 3-6 months
Secondary outcomes (5)
Effect of type of bleed on initial dose, dosing interval and total dose
Measured: after 3-6 months
Effect of initial dose and dosing interval on total dose, time to first perceived improvement, time to perceived bleed resolution
Measured: after 3-6 months
Effect of time to first dose on dosing interval and total dose
Measured: after 3-6 months
Effect of bleeds and drug administration time on planned daily activities
Measured: after 3-6 months
Relationship of dose and dosing intervals to reported SAEs
Measured: after 3-6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2008 (estimated)
- Study startJun 2008
- Primary completionJul 2009
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Bleeding DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00710619
- Sponsor's protocol ID
- F7HAEM-1965
- Acronym
- DOSE
- Status last verified
- Nov 2016
- First submitted
- 3 Jul 2008
- Last updated
- 17 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 23551918
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00710619
- Acronym / sponsor ID
- DOSE / F7HAEM-1965
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Jun 2008
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 4 Jul 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | – | Drug: activated recombinant human factor VII, Drug: Feiba VH |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male subjects with congenital haemophilia A or B with inhibitors with spontaneous bleeds requiring on-demand treatment
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| activated recombinant human factor VIIThis is an observation of how patients/caregivers dose bypassing agents at home for haemophilia with inhibitors | Drug | A |
| Feiba VHThis is an observation of how patients/caregivers dose bypassing agents at home for hemophilia with inhibitors | Drug | A |
Primary outcome
Characterisation of dose and dosing intervals for each bleeding episode
Time frame: after 3-6 months
5 secondary outcomes
Effect of type of bleed on initial dose, dosing interval and total dose
Time frame: after 3-6 months
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
Effect of time to first dose on dosing interval and total dose
Time frame: after 3-6 months
Effect of bleeds and drug administration time on planned daily activities
Time frame: after 3-6 months
Relationship of dose and dosing intervals to reported SAEs
Time frame: after 3-6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2016.
- Derived from this study: PubMed 23551918Gruppo 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.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterisation of dose and dosing intervals for each bleeding episode (time frame: after 3-6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00710619, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2016.