Hemophilia BPhase 4NCT01128881
IMMUNINE Pre-Treatment Study
A Phase 4 study testing Factor IX Concentrate (purified, virus-inactivated) in people with Hemophilia B, run by Baxalta now part of Shire at 18 sites in 10 countries.
Trial summary
The primary objective of this study is to prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors over a period of approximately 20 to 50 exposure days while receiving prophylactic treatment in up to 50 previously treated patients (PTPs) aged 12-64 years and approximately 20 pediatric PTPs up to… Read more about this study
- Who is it for?
- Ages Up to 64 years
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Factor IX Concentrate (purified, virus-inactivated)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 18 sitesin 10 countries
- Study dates
- 31 May 2010to 28 Aug 2012
- Phase
- Phase 4
- 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 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject is up to 64 years old at the time of screening
- Subject and/or legal representative has/have provided signed informed consent
- Subject has severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory
- Subject is previously treated with plasma-derived or recombinant FIX concentrate(s), cryoprecipitate or fresh frozen plasma (FFP) for approximately 100-150 exposure days (EDs) if >= 6 years old, or 20-50 EDs if < 6 years old, and is planned to enter BAX326 study 250901. The number of EDs are derived from the subject's treatment regimen and his/her bleeding pattern
- Subject is willing to receive prophylactic treatment for the duration of the study
- Subject is immunocompetent as evidenced by a CD4 count >= 200 cells/mm(3)
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- Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/μL ~ < 400,000 copies/mL
- Female subject of childbearing potential, presents with a negative serum pregnancy test, and agrees to employ adequate birth control measures for the duration of the study
- Subject is willing and able to comply with the requirements of the protocol
Who cannot join
- The subject has a detectable factor IX inhibitor at screening, with a titer >= 0.6 Bethesda Units (BU) as determined by the Nijmegen modification of the Bethesda assay in the central laboratory
- The subject has a history of factor IX inhibitors with a titer >= 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening
- The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to factor IX concentrate(s)
- The subject has a known hypersensitivity to hamster proteins
- The subject has evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
- The subject is scheduled for elective surgery, unless the surgery is medically required within the anticipated study period
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- The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal)
- The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) exceeding the upper limit of normal (ULN), hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices
- The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels >= 5 times the upper limit of normal. During the study, subjects with chronic hepatitis B or C may have fluctuations of up to 5 times the upper limit of normal but will not require discontinuation.
- The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B
- The subject's platelet count is < 100,000/mL
- The subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject's safety or compliance
- The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug other than anti-retroviral chemotherapy (eg, α-interferon, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day)
- The subject is unwilling to consider further participation in BAX 326 (rFIX) pivotal study 250901 or BAX 326 pediatric study
- The subject has participated in another investigational study within 30 days of enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study
- The subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
Trial locations and contacts
18 sites in 10 countries.
18 sites in 10 countries
Poland4 sites
Hematology and Transplantology Clinic, University Clinic Centre - Medical University Hospital
Gdansk, 80-952
Medical College of the Jagiellonian University
Krakow, 31-501
Medical University Lodz, Department of Hematology
Lodz, 93-510
Institute of Haematology and Transfusion
Warsaw, 02-776
Argentina2 sites
Hospital JR Vidal (Servicio de Hemotologie - Area de Investiagacion
Corrientes, 3400
Instituto de Hemofilia y Medicina Clinica Rubén Dávoli
Rosario, 2000
Brazil2 sites
Hospital do apoio de Brasilia
Distrito Federal, 72620-000
UNIFESP - Universidade Estadual de Sáo Paulo
Sáo Paulo, 040024-002
Chile2 sites
Hospital Dr. Sotero del Rio
Santiago
Hospital san José, Centro de Hemofilia
Santiago
Romania2 sites
Prof. Dr. C. T. Nicolau National Institute for Transfusional Hematology
Bucharest, 11156
Louis Turcanu Emergency Clinical Children´s Hospital
Timișoara
Russia2 sites
Hematology Research Center RAMS, Department of Hemophilia and Other Coagulopathies
Moscow, 125157
Republican Center for Hemophilia Treatment, Outpatient Clinic No. 37
Saint Petersburg, 195213
Ukraine1 site
State Institution "Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine"
Lviv, 79044
About this study
Official title: IMMUNINE - Purified Factor IX Concentrate Virus-Inactivated: A Phase 4, Prospective, Open-label Multicenter Study to Prospectively Document the Exposure of IMMUNINE and to Monitor FIX Inhibitors in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B Who Are Planned to Enter BAX 326 Study 250901 to Investigate a New Recombinant FIX Concentrate
The primary objective of this study is to prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors over a period of approximately 20 to 50 exposure days while receiving prophylactic treatment in up to 50 previously treated patients (PTPs) aged 12-64 years and approximately 20 pediatric PTPs up to 11 years of age with severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B who are planned to enter BAX326 study 250901, provided all eligibility criteria are met.
In addition, this study will evaluate the efficacy, safety, immunogenicity, thrombogenicity, and health-related quality of life (HR QoL) of these subjects.
- Conditions
- Hemophilia B
- Being tested
- Factor IX Concentrate (purified, virus-inactivated)
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Factor IX Concentrate (purified, virus-inactivated)Biological
Also called IMMUNINE
Intravenous injection/infusion; dose for prophylaxis: 20-40 IU/kg bodyweight, twice weekly (which may be adjusted to the subject´s bleeding pattern and lifestyle); dose for bleeding episodes and surgery: according to the Summary of Product Characteristics and the Product Information Leaflet of the respective country.
| Study group | Role | What they receive |
|---|---|---|
| IMMUNINE | Experimental | Not described Receives: Biological: Factor IX Concentrate (purified, virus-inactivated) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemostatic Efficacy
- Number of IMMUNINE infusions required to achieve adequate hemostasis for each bleeding episode
- Overall hemostatic efficacy rating of IMMUNINE for all bleeding episodes (scale of excellent, good, fair, none)
- Annualized bleeding rate
- Consumption of IMMUNINE
- Number of infusions per month and per year (prophylaxis and on-demand)
- Weight-adjusted consumption of IMMUNINE per event (prophylaxis, on-demand), per month and per year
Measured: 28 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2010 (estimated)
- Study start31 May 2010
- Primary completion28 Aug 2012
- Study completion28 Aug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01128881
- Sponsor's protocol ID
- 050901
- Other IDs
- 2009-016719-39, 2009
- Status last verified
- Apr 2021
- First submitted
- 7 May 2010
- Last updated
- 23 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IMMUNINE - Purified Factor IX Concentrate Virus-Inactivated: A Phase 4, Prospective, Open-label Multicenter Study to Prospectively Document the Exposure of IMMUNINE and to Monitor FIX Inhibitors in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B Who Are Planned to Enter BAX 326 Study 250901 to Investigate a New Recombinant FIX Concentrate
- ClinicalTrials.gov ID
- NCT01128881
- Acronym / sponsor ID
- 050901
- Conditions
- Hemophilia B
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 31 May 2010 (actual)
- Primary completion
- 28 Aug 2012 (actual)
- Study completion
- 28 Aug 2012 (actual)
- First posted
- 24 May 2010 (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 prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors over a period of approximately 20 to 50 exposure days while receiving prophylactic treatment in up to 50 previously treated patients (PTPs) aged 12-64 years and approximately 20 pediatric PTPs up to 11 years of age with severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B who are planned to enter BAX326 study 250901, provided all eligibility criteria are met.
In addition, this study will evaluate the efficacy, safety, immunogenicity, thrombogenicity, and health-related quality of life (HR QoL) of these subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IMMUNINE | Experimental | Biological: Factor IX Concentrate (purified, virus-inactivated) |
Who may qualify
- Ages
- Up to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Subject is up to 64 years old at the time of screening
- Subject and/or legal representative has/have provided signed informed consent
- Subject has severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory
- Subject is previously treated with plasma-derived or recombinant FIX concentrate(s), cryoprecipitate or fresh frozen plasma (FFP) for approximately 100-150 exposure days (EDs) if >= 6 years old, or 20-50 EDs if < 6 years old, and is planned to enter BAX326 study 250901. The number of EDs are derived from the subject's treatment regimen and his/her bleeding pattern
- Subject is willing to receive prophylactic treatment for the duration of the study
Key exclusion criteria (16 listed)
- The subject has a detectable factor IX inhibitor at screening, with a titer >= 0.6 Bethesda Units (BU) as determined by the Nijmegen modification of the Bethesda assay in the central laboratory
- The subject has a history of factor IX inhibitors with a titer >= 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening
- The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to factor IX concentrate(s)
- The subject has a known hypersensitivity to hamster proteins
- The subject has evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
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 |
|---|---|---|
| Factor IX Concentrate (purified, virus-inactivated)Also: IMMUNINEIntravenous injection/infusion; dose for prophylaxis: 20-40 IU/kg bodyweight, twice weekly (which may be adjusted to the subject´s bleeding pattern and lifestyle); dose for bleeding episodes and surgery: according to the Summary of Product Characteristics and the Product Information Leaflet of the respective country. | Biological | IMMUNINE |
Primary outcome
Hemostatic Efficacy
Time frame: 28 months
* Number of IMMUNINE infusions required to achieve adequate hemostasis for each bleeding episode * Overall hemostatic efficacy rating of IMMUNINE for all bleeding episodes (scale of excellent, good, fair, none) * Annualized bleeding rate * Consumption of IMMUNINE * Number of infusions per month and per year (prophylaxis and on-demand) * Weight-adjusted consumption of IMMUNINE per event (prophylaxis, on-demand), per month and per year
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 23 Apr 2021.
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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
18 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Factor IX Concentrate (purified, virus-inactivated) in people with Hemophilia B, run by Baxalta now part of Shire at 18 sites in 10 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 9 inclusion and 16 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 23 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Poland, Argentina, Brazil, Chile, Romania and 5 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Hemostatic Efficacy (time frame: 28 months). 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 23 Apr 2021.