Hemophilia APhase 2/3PROTECT-VIIINCT01580293
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
A Phase 2/3 study testing BAY94-9027 in people with Hemophilia A, run by Bayer at 59 sites in 20 countries.
Trial summary
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability. Read more about this study
- Who is it for?
- Ages 12 to 65 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
- 145 participants
- Where
- 59 sitesin 20 countries
- Study dates
- 23 Apr 2012to 21 Nov 2019
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male; 12-65 years of age
- Subjects with severe hemophilia A
- Previously treated with factor VIII for a minimum of 150 exposure days
Who cannot join
- Inhibitors to FVIII (current evidence or history)
- Any other inherited or acquired bleeding disorder in addition to Hemophilia A
- Platelet count < 100,000/mm3
- Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine aminotransferase) > 5x upper limit of normal
Trial locations and contacts
59 sites in 20 countries.
59 sites in 20 countries
United States14 sites
Study site
Chicago, Illinois, 60612
Study site
Cincinnati, Ohio, 45229-3039
Study site
Cleveland, Ohio, 44106-6007
Study site
Columbus, Ohio, 43205
Study site
Detroit, Michigan, 48202
Study site
Hershey, Pennsylvania, 17033
Study site
Jacksonville, Florida, 32207
Study site
Miami, Florida, 33136
Study site
Minneapolis, Minnesota, 55455
Study site
Richmond, Virginia, 23298-0155
Study site
Sacramento, California, 95817
Study site
San Diego, California, 92103-8651
Study site
Syracuse, New York, 13210
Study site
Tucson, Arizona, 85724-5024
Japan6 sites
Study site
Hiroshima, 734-8551
Study site
Kashihara, Nara, 634-8522
Study site
Nagoya, Aichi-ken, 466-8560
Study site
Nishinomiya, Hyōgo, 663-8501
Study site
Shinjuku-ku, Tokyo, 160-0023
Study site
Suginami, Tokyo, 167-0035
France4 sites
Study site
Bron, 69677
Study site
Marseille, 13005
Study site
Reims, 51092
Study site
Rennes, 35033
Italy4 sites
Study site
Milan, Lombardy, 20122
Study site
Naples, Campania, 80131
Study site
Rome, Lazio, 00161
Study site
Turin, Piedmont, 10126
Netherlands4 sites
Study site
Amsterdam, 1105 AZ
Study site
Groningen, 9713 GZ
Study site
Maastricht, 6229 HX
Study site
The Hague, 2545 CH
South Korea4 sites
Study site
Busan, Busan Gwang''yeogsi, 49241
Study site
Daejeon, 35233
Study site
Seoul, 03722
Study site
Seoul, 05278
Singapore3 sites
Study site
Singapore, 119228
Study site
Singapore, 169608
Study site
Singapore, 229 899
Taiwan3 sites
Study site
Changhua, 50006
Study site
Taipei, 100
Study site
Taipei, 11217
United Kingdom3 sites
Study site
London, SE1 7EH
Study site
Newcastle upon Tyne, Vale of Glamorgan, the, NE1 4LP
Study site
Sheffield, S10 2JF
Colombia2 sites
Study site
Baranquilla, Atlántico, 080020
Study site
Medellín, Antioquia, 050030
Germany2 sites
Study site
Bonn, North Rhine-Westphalia, 53127
Study site
Heidelberg, Baden-Wurttemberg, 69004
Turkey (Türkiye)2 sites
Study site
Ankara, 06100
Study site
Izmir, 35100
About this study
Official title: A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability.
The current standard treatment for severe haemophilia A is regularly scheduled infusion of FVIII to keep levels high enough to prevent bleeding. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
Read more
In this trial safety and efficacy of a long-acting recombinant factor VIII molecule is evaluated in subjects with severe Hemophilia A.
120-140 patients will receive open label treatment with long-acting rFVIII either on-demand to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic treatment will receive study drug at dosing intervals between once and twice a week depending on their observed bleeding. Patients will attend the treatment centre for routine blood samples and be required to keep an electronic diary.
Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at least 50 exposure days may be eligible for this study.
Read the detailed description
Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion. Those with continued bleeding will remain in treatment arm and have an increase in dose.
Part B-major surgery - optional sub study included to collect information on efficacy of BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment to this sub-study may be independent of participation in main study.
- Conditions
- Hemophilia A
- Being tested
- BAY94-9027
- Keywords
- Hemophilia A, factor VIII, prophylaxis
- 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
Intravenous infusion of BAY94-9027
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds Receives: Biological: BAY94-9027 |
| Arm 2 | Experimental | Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by 2 infusions per week over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED Receives: Biological: BAY94-9027 |
| Arm 3 | Experimental | Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 5 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED Receives: Biological: BAY94-9027 |
| Arm 4 | Experimental | Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 7 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED Receives: Biological: BAY94-9027 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Number of Total Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A, Main Trial
Annualized number of total bleeds was defined as the annualized sum of spontaneous bleeds and trauma bleeds. A participant who had the one-time increase in dose frequency was regarded as rescued. A rescue bleed was a bleed that occured after the dose frequency was increased. Rescue bleeds and periods were not considered for the annualized bleeding rate (ABR).
Measured: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Secondary outcomes (18)
Annualized Number of Joint Bleeds, Trauma, Spontaneous Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A
A participant who had the one-time increase in dose frequency was regarded as rescued. A rescue bleed was a bleed that occured after the dose frequency was increased. Rescue bleeds and periods were not considered for the ABR.
Measured: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Annualized Number of Total Bleeds in On-demand Treatment Arm and in Each Prophylaxis Arm, Part A, Extension
Annualized number of total bleeds was defined as the annualized sum of spontaneous bleeds and trauma bleeds.
Measured: at least 100 total exposure days acquired, median time 3.9 years up to 7 years maximum
Number of Participants Developed Human Coagulation Factor VIII (FVIII) Inhibitor - Part A
FVIII inhibitor testing was done according to the Nijmegen modified Bethesda assay. A positive inhibitor test was defined with a threshold of ≥0.6 Bethesda unit (BU) at the central laboratory.
Measured: Weeks 0 to 36 during Part A
Number of Bleeds Requiring 1, 2 or >= 3 Infusions to Control the Bleed - Part A
Number of bleeds requiring 1, 2 or >= 3 infusions to control the bleeding
Measured: Weeks 0 to 36
Number of Bleeds According to Locations - Part A
Bleed locations were categorised as joint, muscle, skin/mucosa, internal, others and missing.
Measured: Weeks 0 -36
Number of Bleeds Over Time Since Previous Prophylaxis Infusion - Part A
Measured: Weeks 0 to 36
Number of Bleeds According to Participant's Assessment of Response to Treatment - Part A
Response to treatment was assessed by participant as excellent, good, moderate, poor or missing during Part A of the study.
Measured: Weeks 0 to 36 during Part A
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Total Dose Per Kilogram Per Year - Part A
For prophylaxis patients, the dose is related to all infusions.
Measured: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Dose Per Kilogram Per Infusion - Part A
For prophylaxis patients, the dose per infusion related to prophylaxis infusion.
Measured: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Number of Participants Requiring an Increase in Dose Frequency, or Dose Increase, During Weeks 10 to 36 - Part A
Measured: Weeks 10 to 36 during Part A
Number of Surgeries According to Physician's Assessment of Adequacy of Hemostasis in Major Surgery - Part B
Major surgery was defined as any surgical or invasive procedure (elective or emergent) in which the overall bleeding risk was excessive, required a general anesthetic in an individual without a bleeding disorder, penetrated or exposed a major body cavity, resulted in substantial impairment of physical or physiological functions, or required special anatomic knowledge or manipulative skill. Adequacy of hemostasis was assessed as excellent, good, moderate or poor, by the surgeon or interventionalist during Part B of the study.
Measured: Day of surgery
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Dose Per Kilogram Per Infusion for Major Surgery - Part B
Major surgery was defined as any surgical or invasive procedure (elective or emergent) in which the overall bleeding risk was excessive, required a general anesthetic in an individual without a bleeding disorder, penetrated or exposed a major body cavity, resulted in substantial impairment of physical or physiological functions, or required special anatomic knowledge or manipulative skill. Total dose per kilogram per Infusion was expressed in international units per kilogram per infusion (IU/kg/infusion).
Measured: Up to 3 weeks post-surgery during Part B
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Number of Infusions for Major Surgery - Part B
Major surgery was defined as any surgical or invasive procedure (elective or emergent) in which the overall bleeding risk was excessive, required a general anesthetic in an individual without a bleeding disorder, penetrated or exposed a major body cavity, resulted in substantial impairment of physical or physiological functions, or required special anatomic knowledge or manipulative skill.rFVIII usage expressed as number of infusions and IU/kg per year, as well as IU/kg per event (surgery) was assessed by investigator.
Measured: Up to 3 weeks post-surgery during Part B
Maximum Drug Plasma Concentration (Cmax) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
Cmax: Maximum observed drug concentration following an infusion of 60 IU/kg
Measured: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUC) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
AUC: The total area under the plasma concentration versus time curve following an infusion of 60 IU/kg .
Measured: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Terminal Elimination Half Life (t1/2) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
t1/2: Terminal half-life is the time the plasma concentration during terminal phase is halved following an infusion of 60 IU/kg .
Measured: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Overall Human Coagulation Factor VIII (FVIII) Recovery Value by Chromogenic Assay - Part A
Recovery was calculated by the following formula: Recovery = (post-infusion FVIII activity - pre-infusion FVIII activity ) * weight / dose (in IU). Recovery is the increase of FVIII activity after the injection normalized by dose: IU/dl per IU/kg = kg/dL
Measured: Weeks 0 to 36 during Part A
Change From Baseline in Quality of Life by Hemophilia Specific Quality of Life Instrument or Questionnaire for Adults (Haemo-QoL-A) Overall Score at Week 36 - Part A
Quality of life (QoL) was measured by the Haemo-QoL-A overall score, which ranged from 0 (the worst condition) to 100 (the best condition).
Measured: Week 0 (baseline) and Week 36 during Part A
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2012 (estimated)
- Study start23 Apr 2012
- Primary completion13 Jun 2014
- Study completion21 Nov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 145 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
- NCT01580293
- Sponsor's protocol ID
- 13024
- Other IDs
- 2011-005210-11
- Acronym
- PROTECT-VIII
- Status last verified
- Nov 2023
- First submitted
- 28 Mar 2012
- Last updated
- 7 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Baumann A, Piel I, Hucke F, Sandmann S, Hetzel T, Schwarz T. Pharmacokinetics, excretion, distribution, and metabolism of 60-kDa polyethylene glycol used in BAY 94-9027 in rats and its value for human prediction. Eur J Pharm Sci. 2019 Mar 15;130:11-20. doi: 10.1016/j.ejps.2019.01.015. Epub 2019 Jan 14.
PubMed 30654111Lalezari S, Reding MT, Pabinger I, Holme PA, Negrier C, Chalasani P, Shin HJ, Wang M, Tseneklidou-Stoeter D, Maas Enriquez M. BAY 94-9027 prophylaxis is efficacious and well tolerated for up to >5 years with extended dosing intervals: PROTECT VIII extension interim results. Haemophilia. 2019 Nov;25(6):1011-1019. doi: 10.1111/hae.13853. Epub 2019 Oct 17.
PubMed 31621991Reding MT, Ng HJ, Poulsen LH, Eyster ME, Pabinger I, Shin HJ, Walsch R, Lederman M, Wang M, Hardtke M, Michaels LA. Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII. J Thromb Haemost. 2017 Mar;15(3):411-419. doi: 10.1111/jth.13597. Epub 2017 Feb 22.
PubMed 27992112Reding MT, Pabinger I, Holme PA, Maas Enriquez M, Mancuso ME, Lalezari S, Miesbach W, Di Minno G, Klamroth R, Hermans C. Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study. Ther Adv Hematol. 2023 Apr 22;14:20406207231166779. doi: 10.1177/20406207231166779. eCollection 2023.
PubMed 37113811
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT01580293
- Acronym / sponsor ID
- PROTECT-VIII / 13024
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, factor VIII, prophylaxis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 145 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 23 Apr 2012 (actual)
- Primary completion
- 13 Jun 2014 (actual)
- Study completion
- 21 Nov 2019 (actual)
- First posted
- 19 Apr 2012 (estimated)
- Results
- Posted 6 Dec 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability.
The current standard treatment for severe haemophilia A is regularly scheduled infusion of FVIII to keep levels high enough to prevent bleeding. 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 evaluated in subjects with severe Hemophilia A.
120-140 patients will receive open label treatment with long-acting rFVIII either on-demand to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic treatment will receive study drug at dosing intervals between once and twice a week depending on their observed bleeding. Patients will attend the treatment centre for routine blood samples and be required to keep an electronic diary.
Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at least 50 exposure days may be eligible for this study.
Detailed description
Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion. Those with continued bleeding will remain in treatment arm and have an increase in dose.
Part B-major surgery - optional sub study included to collect information on efficacy of BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment to this sub-study may be independent of participation in main study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: BAY94-9027On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds |
| Arm 2 | Experimental | Biological: BAY94-9027Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by 2 infusions per week over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED |
| Arm 3 | Experimental | Biological: BAY94-9027Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 5 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED |
| Arm 4 | Experimental | Biological: BAY94-9027Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 7 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Male; 12-65 years of age
- Subjects with severe hemophilia A
- Previously treated with factor VIII for a minimum of 150 exposure days
Key exclusion criteria (4 listed)
- Inhibitors to FVIII (current evidence or history)
- Any other inherited or acquired bleeding disorder in addition to Hemophilia A
- Platelet count < 100,000/mm3
- Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine aminotransferase) > 5x 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 |
|---|---|---|
| BAY94-9027Intravenous infusion of BAY94-9027 | Biological | Arm 1, Arm 2, Arm 3, Arm 4 |
Primary outcome
Annualized Number of Total Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A, Main Trial
Time frame: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Annualized number of total bleeds was defined as the annualized sum of spontaneous bleeds and trauma bleeds. A participant who had the one-time increase in dose frequency was regarded as rescued. A rescue bleed was a bleed that occured after the dose frequency was increased. Rescue bleeds and periods were not considered for the annualized bleeding rate (ABR).
18 secondary outcomes
Annualized Number of Joint Bleeds, Trauma, Spontaneous Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A
Time frame: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Annualized Number of Total Bleeds in On-demand Treatment Arm and in Each Prophylaxis Arm, Part A, Extension
Time frame: at least 100 total exposure days acquired, median time 3.9 years up to 7 years maximum
Number of Participants Developed Human Coagulation Factor VIII (FVIII) Inhibitor - Part A
Time frame: Weeks 0 to 36 during Part A
Number of Bleeds Requiring 1, 2 or >= 3 Infusions to Control the Bleed - Part A
Time frame: Weeks 0 to 36
Number of Bleeds According to Locations - Part A
Time frame: Weeks 0 -36
Number of Bleeds Over Time Since Previous Prophylaxis Infusion - Part A
Time frame: Weeks 0 to 36
Number of Bleeds According to Participant's Assessment of Response to Treatment - Part A
Time frame: Weeks 0 to 36 during Part A
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Total Dose Per Kilogram Per Year - Part A
Time frame: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Dose Per Kilogram Per Infusion - Part A
Time frame: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A
Number of Participants Requiring an Increase in Dose Frequency, or Dose Increase, During Weeks 10 to 36 - Part A
Time frame: Weeks 10 to 36 during Part A
Number of Surgeries According to Physician's Assessment of Adequacy of Hemostasis in Major Surgery - Part B
Time frame: Day of surgery
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Dose Per Kilogram Per Infusion for Major Surgery - Part B
Time frame: Up to 3 weeks post-surgery during Part B
Recombinant Human Factor VIII (rFVIII) Usage Expressed as Number of Infusions for Major Surgery - Part B
Time frame: Up to 3 weeks post-surgery during Part B
Maximum Drug Plasma Concentration (Cmax) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
Time frame: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUC) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
Time frame: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Terminal Elimination Half Life (t1/2) Following Single and Multiple Doses of BAY94-9027, Chromogenic Assay - Part A
Time frame: Weeks 0 and 36: pre-infusion (0 hours), post-infusion 15, 30 minutes, 1, 3, 6, 8, 24, 48, 72, 96 hours
Overall Human Coagulation Factor VIII (FVIII) Recovery Value by Chromogenic Assay - Part A
Time frame: Weeks 0 to 36 during Part A
Change From Baseline in Quality of Life by Hemophilia Specific Quality of Life Instrument or Questionnaire for Adults (Haemo-QoL-A) Overall Score at Week 36 - Part A
Time frame: Week 0 (baseline) and Week 36 during Part A
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 30654111Baumann A, Piel I, Hucke F, Sandmann S, Hetzel T, Schwarz T. Pharmacokinetics, excretion, distribution, and metabolism of 60-kDa polyethylene glycol used in BAY 94-9027 in rats and its value for human prediction. Eur J Pharm Sci. 2019 Mar 15;130:11-20. doi: 10.1016/j.ejps.2019.01.015. Epub 2019 Jan 14.
- Background: PubMed 31621991Lalezari S, Reding MT, Pabinger I, Holme PA, Negrier C, Chalasani P, Shin HJ, Wang M, Tseneklidou-Stoeter D, Maas Enriquez M. BAY 94-9027 prophylaxis is efficacious and well tolerated for up to >5 years with extended dosing intervals: PROTECT VIII extension interim results. Haemophilia. 2019 Nov;25(6):1011-1019. doi: 10.1111/hae.13853. Epub 2019 Oct 17.
- Background: PubMed 27992112Reding MT, Ng HJ, Poulsen LH, Eyster ME, Pabinger I, Shin HJ, Walsch R, Lederman M, Wang M, Hardtke M, Michaels LA. Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII. J Thromb Haemost. 2017 Mar;15(3):411-419. doi: 10.1111/jth.13597. Epub 2017 Feb 22.
- Results publication: PubMed 37113811Reding MT, Pabinger I, Holme PA, Maas Enriquez M, Mancuso ME, Lalezari S, Miesbach W, Di Minno G, Klamroth R, Hermans C. Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study. Ther Adv Hematol. 2023 Apr 22;14:20406207231166779. doi: 10.1177/20406207231166779. eCollection 2023.
- Click here to find information about studies related to Bayer Healthcare products conducted in EuropeLink 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
59 sites in 20 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing BAY94-9027 in people with Hemophilia A, run by Bayer at 59 sites in 20 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 4 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 7 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in United States, Japan, France, Italy, Netherlands and 15 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Yes. Results are posted in the registry (first posted 6 Dec 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Total Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A, Main Trial (time frame: On-demand: Weeks 0 -36 and Prophylaxis: Weeks 10 - 36 during Part A). 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 NCT01580293, which also lists 4 linked publications.
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 7 Nov 2023.