Hematologic DiseaseNCT00782470
Evaluation of the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Patients With Severe Hemophilia A
An observational study of Hematologic Disease and Blood Coagulation Disorders, run by Bayer at 10 sites in 4 countries.
Trial summary
Understanding how often the bleeding events occur in the subjects who voluntarily decide to switch from prophylaxis to on-demand and in those subjects who remain on prophylaxis. Also look into the consequences of switching treatment in QoL (quality of life), development of target joints, activity level and reasons… Read more about this study
- Who is it for?
- Ages 14 to 29 years, men only
- Conditions the study is for
- Hematologic Disease, Blood Coagulation Disorders
- What is being tested?
- Recombinant Factor VIII (Kogenate, BAY 14-2222)
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 38 participants
- Where
- 10 sitesin 4 countries
- Study dates
- Dec 2007to Nov 2010
- 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 14 to 29 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Cohort 1 - Prospective Prophylaxis Group Cohort 2 - Prospective On-demand Group Cohort 3 - Retrospective On-demand Group
Who may be able to join
- Severe hemophilia A (<2%)
- For subjects who elect staying on Prophylaxis only: Have been on continuous prophylactic treatment for the past 5 years prior to study entry
- For subjects who elect switching to on-demand only: Have been on continuous prophylactic treatment for the past 5 years, but may have been on intermediate or reduced prophylaxis for the 1-12 months prior to study entry
- For subjects currently on-demand: a retrospective arm of subjects who have been on continuous prophylactic treatment for at least 5 years and switched to on-demand treatment between 13 and 24 months prior to study entry
- Current treatment with rFVIII
Who cannot join
- Other known hematological / bleeding disorders other than hemophilia A
- Participating on another study that may have an impact on bleeding or the objectives of this study
- Known alcohol and drug abuse
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
United States7 sites
Study site
Denver, Colorado, 80262
Study site
Duarte, California, 91010
Study site
Fort Worth, Texas, 76104
Study site
Houston, Texas, 77030
Study site
Jefferson City, Missouri, 65109
Study site
Las Vegas, Nevada, 89109
Study site
Peoria, Illinois, 61614
About this study
Official title: A Prospective, Non-interventional, Multi-center, Open-label Study to Evaluate the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Subjects With Severe Hemophilia A Receiving Prophylaxis and On-demand Treatment Regimen
Understanding how often the bleeding events occur in the subjects who voluntarily decide to switch from prophylaxis to on-demand and in those subjects who remain on prophylaxis. Also look into the consequences of switching treatment in QoL (quality of life), development of target joints, activity level and reasons that might influence the desire to switch.
- Conditions
- Hematologic Disease, Blood Coagulation Disorders
- Being tested
- Recombinant Factor VIII (Kogenate, BAY 14-2222)
- Medical subject headings
- Hematologic Diseases, Blood Coagulation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Factor VIII (Kogenate, BAY 14-2222)Behavioural
Subjects electing to stay on the prophylactic treatment (prospective)
Recombinant Factor VIII (Kogenate, BAY 14-2222)Behavioural
Subjects electing to switch to on-demand treatment (prospective)
Recombinant Factor VIII (Kogenate, BAY 14-2222)Behavioural
Subjects remaining on-demand treatment (retrospective)
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Not described Receives: Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) | |
| Group 2 | Not described Receives: Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) | |
| Group 3 | Not described Receives: Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the frequency of all bleeds (spontaneous and trauma) during the study
Measured: End of Study
Secondary outcomes (4)
To evaluate the change from baseline in HRQoL (health-related quality of life)
Measured: End of Study
To evaluate number of patients that want to return to prophylaxis treatment after having switched to on-demand therapy
Measured: End of Study
To evaluate the change from baseline in the Gilbert score
Measured: End of Study
To evaluate the number of target joint development
Measured: End of Study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2008 (estimated)
- Study startDec 2007
- Primary completionNov 2010
- Study completionNov 2010
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 Hematologic DiseaseStudies of the same condition, open and under way.
- More trials run by BayerThe 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
- Cohort
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bayer Study Director, Study director, Bayer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00782470
- Sponsor's protocol ID
- 12749
- Status last verified
- Oct 2014
- First submitted
- 29 Oct 2008
- Last updated
- 3 Nov 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Manco-Johnson MJ, Sanders J, Ewing N, Rodriguez N, Tarantino M, Humphries T; TEEN/TWEN Study Group. Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study. Haemophilia. 2013 Sep;19(5):727-35. doi: 10.1111/hae.12177. Epub 2013 Jun 11.
PubMed 23750875
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Non-interventional, Multi-center, Open-label Study to Evaluate the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Subjects With Severe Hemophilia A Receiving Prophylaxis and On-demand Treatment Regimen
- ClinicalTrials.gov ID
- NCT00782470
- Acronym / sponsor ID
- 12749
- Conditions
- Hematologic Disease, Blood Coagulation Disorders
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Dec 2007
- Primary completion
- Nov 2010 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 31 Oct 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.
Understanding how often the bleeding events occur in the subjects who voluntarily decide to switch from prophylaxis to on-demand and in those subjects who remain on prophylaxis. Also look into the consequences of switching treatment in QoL (quality of life), development of target joints, activity level and reasons that might influence the desire to switch.
Study design
- Observational model
- Cohort
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) |
| Group 2 | – | Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) |
| Group 3 | – | Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222) |
Who may qualify
- Ages
- 14 to 29 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Cohort 1 - Prospective Prophylaxis Group Cohort 2 - Prospective On-demand Group Cohort 3 - Retrospective On-demand Group
Key inclusion criteria (5 listed)
- Severe hemophilia A (<2%)
- For subjects who elect staying on Prophylaxis only: Have been on continuous prophylactic treatment for the past 5 years prior to study entry
- For subjects who elect switching to on-demand only: Have been on continuous prophylactic treatment for the past 5 years, but may have been on intermediate or reduced prophylaxis for the 1-12 months prior to study entry
- For subjects currently on-demand: a retrospective arm of subjects who have been on continuous prophylactic treatment for at least 5 years and switched to on-demand treatment between 13 and 24 months prior to study entry
- Current treatment with rFVIII
Key exclusion criteria (3 listed)
- Other known hematological / bleeding disorders other than hemophilia A
- Participating on another study that may have an impact on bleeding or the objectives of this study
- Known alcohol and drug abuse
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 |
|---|---|---|
| Recombinant Factor VIII (Kogenate, BAY 14-2222)Subjects electing to stay on the prophylactic treatment (prospective) | Behavioural | Group 1 |
| Recombinant Factor VIII (Kogenate, BAY 14-2222)Subjects electing to switch to on-demand treatment (prospective) | Behavioural | Group 2 |
| Recombinant Factor VIII (Kogenate, BAY 14-2222)Subjects remaining on-demand treatment (retrospective) | Behavioural | Group 3 |
Primary outcome
To evaluate the frequency of all bleeds (spontaneous and trauma) during the study
Time frame: End of Study
4 secondary outcomes
To evaluate the change from baseline in HRQoL (health-related quality of life)
Time frame: End of Study
To evaluate number of patients that want to return to prophylaxis treatment after having switched to on-demand therapy
Time frame: End of Study
To evaluate the change from baseline in the Gilbert score
Time frame: End of Study
To evaluate the number of target joint development
Time frame: End of Study
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 3 Nov 2014.
- Results publication: PubMed 23750875Manco-Johnson MJ, Sanders J, Ewing N, Rodriguez N, Tarantino M, Humphries T; TEEN/TWEN Study Group. Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study. Haemophilia. 2013 Sep;19(5):727-35. doi: 10.1111/hae.12177. Epub 2013 Jun 11.
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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.
Bayer Study Director study director
Bayer
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematologic Disease and Blood Coagulation Disorders, run by Bayer at 10 sites in 4 countries.
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 3 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 3 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada, Germany, United Kingdom. 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?
To evaluate the frequency of all bleeds (spontaneous and trauma) during the study (time frame: End of Study). 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 NCT00782470, 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 3 Nov 2014.