Hemophilia APhase 1NCT01599819
BAX 855 Dose-Escalation Safety Study
A Phase 1 study testing Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII in people with Hemophilia A, run by Baxalta now part of Shire at 8 sites in 4 countries.
Trial summary
The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of BAX 855 compared in crossover with ADVATE, and to evaluate the impact of anti-polyethylene… Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 8 sitesin 4 countries
- Study dates
- 30 Sep 2011to 27 Jul 2012
- 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 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The subject is male and is 18 to 65 years of age at the time of screening
- The subject has severe hemophilia A (factor VIII level < 1%)
- The subject was previously treated with plasma-derived factor VIII (FVIII) concentrates or recombinant FVIII for at least 150 exposure days (EDs)
Who cannot join
- The subject has a detectable FVIII inhibitor at screening, with a titer >= 0.6 BU
- The subject has a documented history of FVIII inhibitors with a titer >= 0.4 BU at any time prior to screening
- The subject has a known hypersensitivity towards mouse or hamster proteins or to polyethylene glycol (PEG)
Trial locations and contacts
8 sites in 4 countries.
8 sites in 4 countries
Germany3 sites
Vivantes Klinikum im Friedrichshain, Hemophilia Care Center for Children and Adults
Berlin, 10249
UKGM Uniklinikum Giessen & Marburg
Giessen, 35392
Harrison Clinical Research Deutschland
Munich, 80636
Japan2 sites
Nara Medical University Hospital
Nara, 630-8521
Tokyo Medical University Hospital
Tokyo, 160-0023
United Kingdom2 sites
Quintiles Drug Research Unit at Guy´s Hospital
London, SE1 1YR
Manchester Haemophilia Comprehensive Care Centre, Manchester Royal Infirmary
Manchester, M13 9WL
Bulgaria1 site
Specialized Hospital for Active Treatment "Joan Pavel"
Sofia, 1233
About this study
Official title: BAX 855 (PEGylated Recombinant Factor VIII): A Phase 1, Prospective, Open Label, Cross-Over, Dose-Escalation Study in Previously Treated Patients (PTPs) With Severe (FVIII < 1%) Hemophilia A
The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of BAX 855 compared in crossover with ADVATE, and to evaluate the impact of anti-polyethylene glycol (PEG) antibodies on PK parameters.
- Conditions
- Hemophilia A
- Being tested
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free MethodBiological
Also called ADVATE
1 PK infusion of ADVATE (low dose in Cohort 1, high dose in Cohort 2)
PEGylated Recombinant Factor VIIIBiological
Also called BAX 855
1 PK infusion of BAX 855 (low dose in Cohort 1, high dose in Cohort 2)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Low dose of ADVATE followed by low dose of BAX 855 Receives: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Biological: PEGylated Recombinant Factor VIII |
| Cohort 2 | Experimental | High dose of ADVATE followed by high dose of BAX 855 Receives: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Biological: PEGylated Recombinant Factor VIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serious and non-serious AEs
Measured: 4 weeks after infusion with BAX 855 and ADVATE
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2012 (estimated)
- Study start30 Sep 2011
- Primary completion27 Jul 2012
- Study completion27 Jul 2012
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.
- 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
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baxter Innovations GmbH
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01599819
- Sponsor's protocol ID
- 261101
- Other IDs
- 2011-002011-28
- Status last verified
- Apr 2021
- First submitted
- 15 May 2012
- Last updated
- 5 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.
PubMed 26157075
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- BAX 855 (PEGylated Recombinant Factor VIII): A Phase 1, Prospective, Open Label, Cross-Over, Dose-Escalation Study in Previously Treated Patients (PTPs) With Severe (FVIII < 1%) Hemophilia A
- ClinicalTrials.gov ID
- NCT01599819
- Acronym / sponsor ID
- 261101
- Conditions
- Hemophilia A
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxter Innovations GmbH
- Study start
- 30 Sep 2011 (actual)
- Primary completion
- 27 Jul 2012 (actual)
- Study completion
- 27 Jul 2012 (actual)
- First posted
- 16 May 2012 (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 objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of BAX 855 compared in crossover with ADVATE, and to evaluate the impact of anti-polyethylene glycol (PEG) antibodies on PK parameters.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, Biological: PEGylated Recombinant Factor VIIILow dose of ADVATE followed by low dose of BAX 855 |
| Cohort 2 | Experimental | Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, Biological: PEGylated Recombinant Factor VIIIHigh dose of ADVATE followed by high dose of BAX 855 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- The subject is male and is 18 to 65 years of age at the time of screening
- The subject has severe hemophilia A (factor VIII level < 1%)
- The subject was previously treated with plasma-derived factor VIII (FVIII) concentrates or recombinant FVIII for at least 150 exposure days (EDs)
Key exclusion criteria (3 listed)
- The subject has a detectable FVIII inhibitor at screening, with a titer >= 0.6 BU
- The subject has a documented history of FVIII inhibitors with a titer >= 0.4 BU at any time prior to screening
- The subject has a known hypersensitivity towards mouse or hamster proteins or to polyethylene glycol (PEG)
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 |
|---|---|---|
| Antihemophilic Factor (Recombinant) - Plasma/Albumin Free MethodAlso: ADVATE1 PK infusion of ADVATE (low dose in Cohort 1, high dose in Cohort 2) | Biological | Cohort 1, Cohort 2 |
| PEGylated Recombinant Factor VIIIAlso: BAX 8551 PK infusion of BAX 855 (low dose in Cohort 1, high dose in Cohort 2) | Biological | Cohort 1, Cohort 2 |
Primary outcome
Serious and non-serious AEs
Time frame: 4 weeks after infusion with BAX 855 and ADVATE
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 5 May 2021.
- Derived from this study: PubMed 26157075Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.
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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.
Study Director study director
Takeda
8 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII in people with Hemophilia A, run by Baxalta now part of Shire at 8 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 3 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 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Germany, Japan, United Kingdom, Bulgaria. 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?
Serious and non-serious AEs (time frame: 4 weeks after infusion with BAX 855 and ADVATE). 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 NCT01599819, 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 5 May 2021.