Hemophilia BPhase 1NCT01233440
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
A Phase 1 study testing Recombinant Coagulation Factor IX Albumin Fusion Protein and Plasma derived FIX [pdFIX] in people with Hemophilia B, run by CSL Behring at 20 sites in 6 countries.
Trial summary
The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. The secondary objective of the study is to evaluate the pharmacokinetics parameters, following a single intravenous dose of rIX-FP. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Recombinant Coagulation Factor IX Albumin Fusion Protein and Plasma derived FIX [pdFIX]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 20 sitesin 6 countries
- Study dates
- Oct 2010to Jul 2011
- 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 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male, 12 - 65 years, with body weight ≥ 30 kg and ≤ 120 kg
- Documented severe Hemophilia B (FIX activity of ≤ 2%) or tested by the central laboratory at screening
- Subjects who have received FIX products for > 150 exposure days (EDs) (estimated)
- No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined as two consecutive positive tests - a confirmatory test on a second, separately drawn sample shortly after the previous positive test) and confirmed no detectable FIX inhibitors (negative FIX inhibitor defined as < 0.6 Bethesda Units [BU] by the central laboratory at screening
- Subjects can be treated on-demand or under prophylactic therapy
- Signed Informed Consent/Assent
Who cannot join
- Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein
- Any known congenital or acquired coagulation disorder other than congenital FIX deficiency
- Platelet count < 100,000/µL
- Immunocompromised (CD4 count < 200/mm3), (HIV positive subjects may participate in the study and protease inhibitors and antiviral therapy are permitted, at the discretion of the Investigator)
- Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment
- Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 times (x) the upper limit of normal (ULN)
Show all 12Show fewer
- Serum creatinine > 2 x ULN
- Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months prior to enrollment
- Use of an Investigational Medicinal Product (IMP) within 30 days prior to the first rIX-FP administration
- Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to study entry
- Subject currently on a dose and/or regimen of FIX that would preclude participation in the study due to possible increased risk of bleeding because of the requirement to withhold treatment during the PK sampling period
- Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance
Trial locations and contacts
20 sites in 6 countries.
20 sites in 6 countries
Italy7 sites
Study Site
Catania
Study Site
Florence
Study Site
Genova
Study site
Milan
Study Site
Naples
Study Site
Parma
Study Site
Vicenza
France4 sites
Study site
Le Kremlin-Bicêtre
Study Site
Lyon
Study site
Nantes
Study site
Paris
Germany4 sites
Study Site
Berlin
Study Site
Hamburg
Study Site
Hanover
Study site
Münster
About this study
Official title: An Open-label, Multicenter, Dose-Escalation Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. The secondary objective of the study is to evaluate the pharmacokinetics parameters, following a single intravenous dose of rIX-FP.
Read the detailed description
This study is comprised of both a rIX-FP dose-escalation safety segment (25, 50 and 75 IU/kg of rIX-FP), and PK evaluation of rIX-FP after a single dose of 50 IU/kg, as well as PK evaluation after a single dose of 50 IU/kg of the previously given Factor IX (FIX) product (recombinant FIX [rFIX] or plasma derived FIX [pdFIX]) which is used as the reference product.
- Conditions
- Hemophilia B
- Being tested
- Recombinant Coagulation Factor IX Albumin Fusion Protein, Plasma derived FIX [pdFIX]
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Coagulation Factor IX Albumin Fusion ProteinBiological
Single dose of 25, 50 or 75 IU/kg of rIX-FP, given as intravenous infusion
Plasma derived FIX [pdFIX]Biological
Single dose of 50 IU/kg of reference product, given as intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 25 IU/kg dose Receives: Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein |
| Cohort 2 | Experimental | 50 IU/kg dose Receives: Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein; Biological: Plasma derived FIX [pdFIX] |
| Cohort 3 | Experimental | 75 IU/kg dose Receives: Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of adverse events (AEs)
Measured: up to 14 days after drug administration
Frequency of serious adverse events (SAEs)
Measured: up to 28 days after drug administration
Occurrence of inhibitor against FIX
Measured: up to 28 days after drug administration
Occurrence of antibodies against rIX-FP
Measured: up to 28 days after drug administration
Secondary outcomes (5)
AUC to the last sample with quantifiable drug concentration (AUC0-t)
Following 50 IU/kg rIX-FP infusion
Measured: From time of dosing up to 7 days after the dose
AUC extrapolated to infinity (AUCt-∞)
Following 50 IU/kg rIX-FP infusion
Measured: From time of dosing up to 7 days after the dose
Half-life (t1/2)
Following 50 IU/kg rIX-FP infusion
Measured: From time of dosing up to 7 days after the dose
Incremental recovery (IU/mL/IU/kg)
Defined as FIX activity (IU/mL) obtained 30 minutes following infusion, per dose of (IU/kg) infusion.
Measured: From time of dosing up to 7 days after the dose
Clearance
Following 50 IU/kg rIX-FP infusion
Measured: From time of dosing up to 7 days after the dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2010 (estimated)
- Study startOct 2010
- Primary completionJul 2011
- Study completionJul 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by CSL BehringThe 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Iris Jacobs, MD, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01233440
- Sponsor's protocol ID
- CSL654_2001
- Other IDs
- 1508, 2010-018477-38
- Status last verified
- Jan 2012
- First submitted
- 2 Nov 2010
- Last updated
- 31 Jan 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Koopman SF, Cnossen MH, Mathot RAA; OPTI-CLOT study group and SYMPHONY consortium. Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis. Clin Pharmacol Ther. 2025 Oct;118(4):831-840. doi: 10.1002/cpt.3724. Epub 2025 May 19.
PubMed 40386832Santagostino E, Negrier C, Klamroth R, Tiede A, Pabinger-Fasching I, Voigt C, Jacobs I, Morfini M. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012 Sep 20;120(12):2405-11. doi: 10.1182/blood-2012-05-429688. Epub 2012 Aug 2.
PubMed 22859609
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter, Dose-Escalation Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
- ClinicalTrials.gov ID
- NCT01233440
- Acronym / sponsor ID
- CSL654_2001
- Conditions
- Hemophilia B
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Oct 2010
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 3 Nov 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 the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. The secondary objective of the study is to evaluate the pharmacokinetics parameters, following a single intravenous dose of rIX-FP.
Detailed description
This study is comprised of both a rIX-FP dose-escalation safety segment (25, 50 and 75 IU/kg of rIX-FP), and PK evaluation of rIX-FP after a single dose of 50 IU/kg, as well as PK evaluation after a single dose of 50 IU/kg of the previously given Factor IX (FIX) product (recombinant FIX [rFIX] or plasma derived FIX [pdFIX]) which is used as the reference product.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein25 IU/kg dose |
| Cohort 2 | Experimental | Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein, Biological: Plasma derived FIX [pdFIX]50 IU/kg dose |
| Cohort 3 | Experimental | Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein75 IU/kg dose |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male, 12 - 65 years, with body weight ≥ 30 kg and ≤ 120 kg
- Documented severe Hemophilia B (FIX activity of ≤ 2%) or tested by the central laboratory at screening
- Subjects who have received FIX products for > 150 exposure days (EDs) (estimated)
- No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined as two consecutive positive tests - a confirmatory test on a second, separately drawn sample shortly after the previous positive test) and confirmed no detectable FIX inhibitors (negative FIX inhibitor defined as < 0.6 Bethesda Units [BU] by the central laboratory at screening
- Subjects can be treated on-demand or under prophylactic therapy
Key exclusion criteria (12 listed)
- Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein
- Any known congenital or acquired coagulation disorder other than congenital FIX deficiency
- Platelet count < 100,000/µL
- Immunocompromised (CD4 count < 200/mm3), (HIV positive subjects may participate in the study and protease inhibitors and antiviral therapy are permitted, at the discretion of the Investigator)
- Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment
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 |
|---|---|---|
| Recombinant Coagulation Factor IX Albumin Fusion ProteinSingle dose of 25, 50 or 75 IU/kg of rIX-FP, given as intravenous infusion | Biological | Cohort 1, Cohort 2, Cohort 3 |
| Plasma derived FIX [pdFIX]Single dose of 50 IU/kg of reference product, given as intravenous infusion | Biological | Cohort 2 |
Primary outcome
Frequency of adverse events (AEs)
Time frame: up to 14 days after drug administration
Frequency of serious adverse events (SAEs)
Time frame: up to 28 days after drug administration
Occurrence of inhibitor against FIX
Time frame: up to 28 days after drug administration
Occurrence of antibodies against rIX-FP
Time frame: up to 28 days after drug administration
5 secondary outcomes
AUC to the last sample with quantifiable drug concentration (AUC0-t)
Time frame: From time of dosing up to 7 days after the dose
AUC extrapolated to infinity (AUCt-∞)
Time frame: From time of dosing up to 7 days after the dose
Half-life (t1/2)
Time frame: From time of dosing up to 7 days after the dose
Incremental recovery (IU/mL/IU/kg)
Time frame: From time of dosing up to 7 days after the dose
Clearance
Time frame: From time of dosing up to 7 days after the dose
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 31 Jan 2012.
- Derived from this study: PubMed 40386832Koopman SF, Cnossen MH, Mathot RAA; OPTI-CLOT study group and SYMPHONY consortium. Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis. Clin Pharmacol Ther. 2025 Oct;118(4):831-840. doi: 10.1002/cpt.3724. Epub 2025 May 19.
- Derived from this study: PubMed 22859609Santagostino E, Negrier C, Klamroth R, Tiede A, Pabinger-Fasching I, Voigt C, Jacobs I, Morfini M. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012 Sep 20;120(12):2405-11. doi: 10.1182/blood-2012-05-429688. Epub 2012 Aug 2.
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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.
Iris Jacobs, MD study director
CSL Behring
20 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Recombinant Coagulation Factor IX Albumin Fusion Protein and Plasma derived FIX [pdFIX] in people with Hemophilia B, run by CSL Behring at 20 sites in 6 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 6 inclusion and 12 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 31 Jan 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Italy, France, Germany, Spain, Austria and 1 more country. 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?
Frequency of adverse events (AEs) (time frame: up to 14 days after drug administration). The study lists 4 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 NCT01233440, which also lists 2 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 31 Jan 2012.