Hemophilia APhase 3NCT01775618
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
A Phase 3 study testing BAY94-9027 in people with Hemophilia A, run by Bayer at 37 sites in 17 countries.
Trial summary
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. Read more about this study
- Who is it for?
- Ages Up to 12 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- BAY94-9027
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 73 participants
- Where
- 37 sitesin 17 countries
- Study dates
- 29 May 2013to 19 Feb 2020
- Phase
- Phase 3
- 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 Up to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males < 12 years of age
- Subjects with severe hemophilia A
- Previously treated with FVIII for > 50 exposure days
Who cannot join
- Subjects with current evidence of or history of inhibitors to FVIII
- Any other inherited or acquired bleeding disorder
- Platelet counts < 100,000/mm^3
- Creatinine > 2x the upper limit of normal
- Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
Trial locations and contacts
37 sites in 17 countries.
37 sites in 17 countries
United States7 sites
Study site
Cincinnati, Ohio, 45229
Study site
Cleveland, Ohio, 44106-6007
Study site
Columbus, Ohio, 43205-2696
Study site
Hershey, Pennsylvania, 17033
Study site
Pensacola, Florida, 32504
Study site
Sacramento, California, 95817
Study site
Salt Lake City, Utah, 84113
United Kingdom4 sites
Study site
Bristol, BS2 8AE
Study site
Manchester, M13 9WL
Study site
Newcastle upon Tyne, Tyne and Wear, NE1 4LP
Study site
Sheffield, S10 2TH
Bulgaria3 sites
Study site
Plovdiv, 4002
Study site
Sofia, 1527
Study site
Varna, 9010
Italy3 sites
Study site
Milan, Lombardy, 20122
Study site
Padova, Veneto, 35128
Study site
Palermo, Sicily, 90127
Romania3 sites
Study site
Bucharest, 011026
Study site
Bucharest, 022328
Study site
Timișoara, 300011
Canada2 sites
Study site
Calgary, Alberta, T3B 6A8
Study site
Toronto, Ontario, M5G 1X8
About this study
Official title: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
- Conditions
- Hemophilia A
- Being tested
- BAY94-9027
- Keywords
- Hemophilia A, Factor VIII, Prophylaxis, Pediatric
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAY94-9027Biological
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months
BAY94-9027Biological
Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks
BAY94-9027Biological
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 additional EDs to achieve at least 100 cumulative EDs, or until marketing authorization of the drug
| Study group | Role | What they receive |
|---|---|---|
| Main study | Experimental | Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25-60 international units/kilogram (IU/kg) twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an intravenous (IV) infusion as per clinical needs of each subject up to at least 50 exposure days (EDs) and a minimum of at least 6 months. Receives: Biological: BAY94-9027 |
| Part 2 (Expansion group) | Experimental | Participants were administered with BAY94-9027 at a dose of 25-60 IU/kg twice per week for prophylaxis for 12 weeks. Receives: Biological: BAY94-9027 |
| Extension study | Experimental | Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25- 60 IU/kg twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an IV infusion as per clinical needs of each subject for at least 50 EDs or until marketing authorization of the drug. Receives: Biological: BAY94-9027 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized number of all bleeds
Measured: At least 50 exposure days (ED) over 6 months, on average 245 days
Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
Pharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)
Measured: Pre-dose to 72 hours post-dose
Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
Measured: At least 50 exposure days (ED) over 6 months, on average 245 days
Characterization of a potential immune response
Measured: 12 weeks
Inhibitor development in the extension study
Measured: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary outcomes (3)
Inhibitor development in the main study
Measured: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
Assessment of incremental recovery in main study
Measured: At least 50 exposure days (ED) over 6 months, on average 245 days
Number of participants with adverse events as a measure of safety and tolerability
Measured: From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2013 (estimated)
- Study start29 May 2013
- Primary completion19 Mar 2015
- Study completion19 Feb 2020
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 Hemophilia AStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 73 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
- NCT01775618
- Sponsor's protocol ID
- 15912
- Other IDs
- 2012-004434-42
- Status last verified
- Aug 2020
- First submitted
- 23 Jan 2013
- Last updated
- 21 Aug 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Mancuso ME, Biss T, Fischer K, Maas Enriquez M, Steele M, Wang M, Tseneklidou-Stoeter D, Ahuja S, Kenet G. PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. Haemophilia. 2021 May;27(3):434-444. doi: 10.1111/hae.14294. Epub 2021 Mar 16.
PubMed 33724632
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT01775618
- Acronym / sponsor ID
- 15912
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Factor VIII, Prophylaxis, Pediatric
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 29 May 2013 (actual)
- Primary completion
- 19 Mar 2015 (actual)
- Study completion
- 19 Feb 2020 (actual)
- First posted
- 25 Jan 2013 (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.
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Main study | Experimental | Biological: BAY94-9027Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25-60 international units/kilogram (IU/kg) twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an intravenous (IV) infusion as per clinical needs of each subject up to at least 50 exposure days (EDs) and a minimum of at least 6 months. |
| Part 2 (Expansion group) | Experimental | Biological: BAY94-9027Participants were administered with BAY94-9027 at a dose of 25-60 IU/kg twice per week for prophylaxis for 12 weeks. |
| Extension study | Experimental | Biological: BAY94-9027Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25- 60 IU/kg twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an IV infusion as per clinical needs of each subject for at least 50 EDs or until marketing authorization of the drug. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Males < 12 years of age
- Subjects with severe hemophilia A
- Previously treated with FVIII for > 50 exposure days
Key exclusion criteria (5 listed)
- Subjects with current evidence of or history of inhibitors to FVIII
- Any other inherited or acquired bleeding disorder
- Platelet counts < 100,000/mm^3
- Creatinine > 2x the upper limit of normal
- Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
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 |
|---|---|---|
| BAY94-9027Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months | Biological | Main study |
| BAY94-9027Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks | Biological | Part 2 (Expansion group) |
| BAY94-9027Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 additional EDs to achieve at least 100 cumulative EDs, or until marketing authorization of the drug | Biological | Extension study |
Primary outcome
Annualized number of all bleeds
Time frame: At least 50 exposure days (ED) over 6 months, on average 245 days
Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
Time frame: Pre-dose to 72 hours post-dose
Pharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)
Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
Time frame: At least 50 exposure days (ED) over 6 months, on average 245 days
Characterization of a potential immune response
Time frame: 12 weeks
Inhibitor development in the extension study
Time frame: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
3 secondary outcomes
Inhibitor development in the main study
Time frame: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
Assessment of incremental recovery in main study
Time frame: At least 50 exposure days (ED) over 6 months, on average 245 days
Number of participants with adverse events as a measure of safety and tolerability
Time frame: From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days)
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 21 Aug 2020.
- Derived from this study: PubMed 33724632Mancuso ME, Biss T, Fischer K, Maas Enriquez M, Steele M, Wang M, Tseneklidou-Stoeter D, Ahuja S, Kenet G. PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. Haemophilia. 2021 May;27(3):434-444. doi: 10.1111/hae.14294. Epub 2021 Mar 16.
- Click here to find information about studies related to Bayer Healthcare products conducted in Europe.Link 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.
Bayer Study Director study director
Bayer
37 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BAY94-9027 in people with Hemophilia A, run by Bayer at 37 sites in 17 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 3 inclusion and 5 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 21 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, United Kingdom, Bulgaria, Italy, Romania and 12 more countries. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Annualized number of all bleeds (time frame: At least 50 exposure days (ED) over 6 months, on average 245 days). The study lists 5 primary outcomes in all. 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 NCT01775618, 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 21 Aug 2020.