Hemophilia APhase 1NCT00629837
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
A Phase 1 study testing Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) and Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 2 sites in United States.
Trial summary
The primary objective of this study is to determine the pharmacokinetic profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/Kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs aged 12 to… Read more about this study
- Who is it for?
- Ages 12 to 60 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) and Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2005to Mar 2006
- Phase
- Phase 1
- 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 12 to 60 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males aged 12 to 60 years
- Hemophilia A with plasma FVIII level less than 1% (severe hemophilia)
- No history of FVIII inhibitor antibody formation and no current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay (< 0.6 Nijmegen Bethesda Units [N.B.U.]/mL)
- No signs or symptoms of an acute bleeding episode on the day of infusion
- Four or more days without treatment with FVIII prior to the day of infusion
- Subject (or the subject's legal representative) must provide written informed consent and authorization of use and disclosure of Protected Health Information (PHI)
- Subjects must have been previously treated with FVIII concentrate for a total of at least 200 exposure days, including 20 exposure days in the previous 12 months. Previous treatment can have been with any type of rFVIII or plasma-derived FVIII concentrate
Who cannot join
- Individuals with abnormal renal function (serum creatinine concentrations greater than 1.3 mg/dL) or active hepatic disease (persistent aspartate aminotransferase [AST] or alanine aminotransferase [ALT] increases to greater than five times the upper limit of normal).
- Individuals with anemia, as defined by hemoglobin level less than 12 g/dL
- Any individual with a past history of severe reaction(s) to FVIII products
- Any individual on interferon treatment or who has received interferon within the previous 3 months
- Any individual with thrombocytopenia (platelets greater than or equal to 100,000 cells/mm3) or known hematologic/bleeding problems other than hemophilia A
- Any individual who is receiving or has received other experimental drugs within 3 months prior to study entry
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- Any individual with known dislipidemic disease or actively taking cholesterol lowering drugs for the treatment of hypercholesterolemia or hyperlipidemia (e.g., statins, cholesterol absorption inhibitors, bile acid sequestrants, nicotinic acid or fibrates) or individuals taking anaesthetic drugs
- Any individual who requires pre-medication for FVIII infusions (e.g., antihistamines)
- Any individual with high blood pressure (defined as diastolic blood pressure great than or equal to 100 mm/Hg)
- Any patient who cannot forego FVIII treatment for at least 4 days prior to study entry or between study infusions due to a need for more frequent prophylactic treatment because of a pre-existing medical condition
- Any patient with known allergy or severe reactions to liposomes or PEG
- Individuals with any other known disease affecting hemostasis besides hemophilia A
- Any patient who is not suitable for participation in this trial for any reason, according to the Investigator
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Study site
Davis, California, 95616
Study site
Orange, California, 92868-3974
About this study
Official title: A Randomized, Double-blind, Cross-over Study to Determine the Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980 in Previously Treated Patients With Severe Hemophilia A
The primary objective of this study is to determine the pharmacokinetic profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/Kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs aged 12 to 60 years with severe hemophilia A.
- Conditions
- Hemophilia A
- Being tested
- Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Keywords
- FVIII, Long acting, pk
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)Biological
Low dose of BAY 79-4980 [13mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU).
Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)Biological
High dose of BAY 79-4980 [22mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU)
Recombinant Factor VIII (Kogenate FS, BAY14-2222)Biological
rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to low dose of BAY 79-4980 [13mg of liposomes/kg]
Recombinant Factor VIII (Kogenate FS, BAY14-2222)Biological
rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to high dose of BAY 79-4980 [22mg of liposomes/kg]
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Not described Receives: Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) |
| Arm 2 | Experimental | Not described Receives: Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) |
| Arm 3 | Active comparator | Not described Receives: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
| Arm 4 | Active comparator | Not described Receives: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the pk profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs
Measured: 6 weeks
Secondary outcomes (5)
To determine the infusion tolerability of both BAY 79-4980 doses, by evaluation of vital signs and adverse events
Measured: 6 weeks
To determine the safety of both BAY 79-4980 doses by measuring the effects on laboratory parameters - especially the lipid profile and adverse events
Measured: 6 weeks
To determine the pk characteristics of liposomes - esp body clearance by measuring the major liposome component 1-palmitoyl-2-oleoyl-sn-glycerol-3-phosphocholine (POPC) and the non-biological liposome component, MPEG 2000 DSPE, as surrogate marker
Measured: 6 weeks
To determine the activity of rFVIII over time (as determined by thrombin generation assay and the rotation thromboelastography [RoTEG] assay) for both doses of BAY 79-4980 compared to rFVIII-FS
Measured: 6 weeks
Additional analyses of the number and timing of spontaneous bleeds after each study treatment will be assessed
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2008 (estimated)
- Study startSep 2005
- Study completionMar 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 27 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
- NCT00629837
- Sponsor's protocol ID
- 11876
- Other IDs
- LipLong
- Status last verified
- Nov 2014
- First submitted
- 27 Feb 2008
- Last updated
- 18 Nov 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Cross-over Study to Determine the Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980 in Previously Treated Patients With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT00629837
- Acronym / sponsor ID
- 11876
- Conditions
- Hemophilia A
- Keywords
- FVIII, Long acting, pk
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Sep 2005
- Study completion
- Mar 2006 (actual)
- First posted
- 6 Mar 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.
The primary objective of this study is to determine the pharmacokinetic profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/Kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs aged 12 to 60 years with severe hemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) |
| Arm 2 | Experimental | Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) |
| Arm 3 | Active comparator | Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
| Arm 4 | Active comparator | Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Males aged 12 to 60 years
- Hemophilia A with plasma FVIII level less than 1% (severe hemophilia)
- No history of FVIII inhibitor antibody formation and no current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay (< 0.6 Nijmegen Bethesda Units [N.B.U.]/mL)
- No signs or symptoms of an acute bleeding episode on the day of infusion
- Four or more days without treatment with FVIII prior to the day of infusion
Key exclusion criteria (13 listed)
- Individuals with abnormal renal function (serum creatinine concentrations greater than 1.3 mg/dL) or active hepatic disease (persistent aspartate aminotransferase [AST] or alanine aminotransferase [ALT] increases to greater than five times the upper limit of normal).
- Individuals with anemia, as defined by hemoglobin level less than 12 g/dL
- Any individual with a past history of severe reaction(s) to FVIII products
- Any individual on interferon treatment or who has received interferon within the previous 3 months
- Any individual with thrombocytopenia (platelets greater than or equal to 100,000 cells/mm3) or known hematologic/bleeding problems other than hemophilia A
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 |
|---|---|---|
| Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)Low dose of BAY 79-4980 [13mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU). | Biological | Arm 1 |
| Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)High dose of BAY 79-4980 [22mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU) | Biological | Arm 2 |
| Recombinant Factor VIII (Kogenate FS, BAY14-2222)rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to low dose of BAY 79-4980 [13mg of liposomes/kg] | Biological | Arm 3 |
| Recombinant Factor VIII (Kogenate FS, BAY14-2222)rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to high dose of BAY 79-4980 [22mg of liposomes/kg] | Biological | Arm 4 |
Primary outcome
To determine the pk profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs
Time frame: 6 weeks
5 secondary outcomes
To determine the infusion tolerability of both BAY 79-4980 doses, by evaluation of vital signs and adverse events
Time frame: 6 weeks
To determine the safety of both BAY 79-4980 doses by measuring the effects on laboratory parameters - especially the lipid profile and adverse events
Time frame: 6 weeks
To determine the pk characteristics of liposomes - esp body clearance by measuring the major liposome component 1-palmitoyl-2-oleoyl-sn-glycerol-3-phosphocholine (POPC) and the non-biological liposome component, MPEG 2000 DSPE, as surrogate marker
Time frame: 6 weeks
To determine the activity of rFVIII over time (as determined by thrombin generation assay and the rotation thromboelastography [RoTEG] assay) for both doses of BAY 79-4980 compared to rFVIII-FS
Time frame: 6 weeks
Additional analyses of the number and timing of spontaneous bleeds after each study treatment will be assessed
Time frame: 6 weeks
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 18 Nov 2014.
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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
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) and Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 2 sites 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 7 inclusion and 13 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 18 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 2 sites 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
To determine the pk profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Nov 2014.