Hemophilia BPhase 1NCT00716716
Phase I/IIa Study of FIXFc in Hemophilia B Patients
A Phase 1 study testing rFIXFc in people with Hemophilia B, run by Bioverativ Therapeutics Inc. at 6 sites in United States.
Trial summary
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- rFIXFc
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 6 sitesUnited States
- Study dates
- Apr 2008to Oct 2009
- 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 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Are previously treated (PTPs) with severe (<2 IU/dL endogenous FIX level) hemophilia B and at least 150 prior documented exposure days to other FIX products.
- Have no prior history of or currently detectable inhibitor defined as > 0.6 Bethesda units by the local lab. A family history of inhibitors will not exclude the patient.
- No prior history of an allergic reaction or anaphylaxis associated with any FIX or IVIG administration.
- No concurrent autoimmune disease.
- At least 7 days since their last dose of FIX (wash-out period).
- Certain laboratory testing criteria and other protocol-defined criteria may apply.
- HIV negative or if HIV positive with a CD4 count ≥ 200 cells/mm3. HIV patients are allowed to receive protease inhibitors per the discretion of the Investigator.
Who cannot join
- Presence of a major bleeding episode on Day 1 of study.
- Any coagulation disorder in addition to hemophilia B.
- A patient currently on a dose and 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 study period.
- A positive d-dimer at screening.
- Documented history of liver cirrhosis.
- Positive for HBsAg and/or positive for hepatitis C antibody, and/or HIV positive with an ALT or AST greater than 5 times upper limit of normal.
- Certain prior illnesses and other protocol-defined criteria.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Brigham & Women's Hospital
Boston, Massachusetts, 02115
University of North Carolina Medical School
Chapel Hill, North Carolina, 27599
RUSH University Medical Center
Chicago, Illinois, 60612
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, 15213
Puget Sound Blood Center
Seattle, Washington, 98104
About this study
Official title: A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg.
Read the detailed description
This study was previously posted by Syntonix Pharmaceuticals, Inc. In January, 2007, sponsorship of the trial was transferred to Biogen Idec. In February, 2017, sponsorship of the trial was transferred to Bioverativ.
- Conditions
- Hemophilia B
- Being tested
- rFIXFc
- Keywords
- Hemophilia B severe, previously treated patients
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rFIXFcDrug
Also called Alprolix, recombinant factor IX fusion protein, BIIB029, FIXFc
As specified in the treatment arm
| Study group | Role | What they receive |
|---|---|---|
| rFIXFc | Experimental | Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg Receives: Drug: rFIXFc |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants experiencing Adverse Events
Measured: Up to 45 days
Secondary outcomes (10)
Time to reach maximum concentration (Tmax)
Measured: Up to 45 days
Maximum concentration (Cmax)
Measured: Up to 45 days
Half-life (t½)
Measured: Up to 45 days
Clearance (CL)
Measured: Up to 45 days
Volume of distribution (Vd)
Measured: Up to 45 days
Area under the curve (AUC)
Measured: Up to 45 days
Mean residence time (MRT)
Measured: Up to 45 days
Incremental recovery (K)
Measured: Up to 45 days
Factor IX protein (FIX) activity
Measured: Up to 45 days
Recombinant (FIXFc) concentration over time curves
Measured: up to 45 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2008 (estimated)
- Study startApr 2008
- Primary completionOct 2009
- Study completionOct 2009
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 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Syntonix Pharmaceuticals, Inc., Swedish Orphan Biovitrum
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Bioverativ Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00716716
- Sponsor's protocol ID
- SYN-FIXFc-07-001
- Status last verified
- Aug 2018
- First submitted
- 14 Jul 2008
- Last updated
- 19 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallen B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood. 2012 Jan 19;119(3):666-72. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22.
PubMed 22110246
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa Safety and Pharmacokinetic Study of Intravenous FIXFc in Previously Treated Hemophilia B Patients
- ClinicalTrials.gov ID
- NCT00716716
- Acronym / sponsor ID
- SYN-FIXFc-07-001
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B severe, previously treated patients
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Bioverativ Therapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Syntonix Pharmaceuticals, Inc., Swedish Orphan Biovitrum
- Study start
- Apr 2008
- Primary completion
- Oct 2009 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 16 Jul 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 the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg.
Detailed description
This study was previously posted by Syntonix Pharmaceuticals, Inc. In January, 2007, sponsorship of the trial was transferred to Biogen Idec. In February, 2017, sponsorship of the trial was transferred to Bioverativ.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rFIXFc | Experimental | Drug: rFIXFcSix intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Are previously treated (PTPs) with severe (<2 IU/dL endogenous FIX level) hemophilia B and at least 150 prior documented exposure days to other FIX products.
- Have no prior history of or currently detectable inhibitor defined as > 0.6 Bethesda units by the local lab. A family history of inhibitors will not exclude the patient.
- No prior history of an allergic reaction or anaphylaxis associated with any FIX or IVIG administration.
- No concurrent autoimmune disease.
- At least 7 days since their last dose of FIX (wash-out period).
Key exclusion criteria (8 listed)
- Presence of a major bleeding episode on Day 1 of study.
- Any coagulation disorder in addition to hemophilia B.
- A patient currently on a dose and 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 study period.
- A positive d-dimer at screening.
- Documented history of liver cirrhosis.
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 |
|---|---|---|
| rFIXFcAlso: Alprolix, recombinant factor IX fusion protein, BIIB029, FIXFcAs specified in the treatment arm | Drug | rFIXFc |
Primary outcome
Number of Participants experiencing Adverse Events
Time frame: Up to 45 days
10 secondary outcomes
Time to reach maximum concentration (Tmax)
Time frame: Up to 45 days
Maximum concentration (Cmax)
Time frame: Up to 45 days
Half-life (t½)
Time frame: Up to 45 days
Clearance (CL)
Time frame: Up to 45 days
Volume of distribution (Vd)
Time frame: Up to 45 days
Area under the curve (AUC)
Time frame: Up to 45 days
Mean residence time (MRT)
Time frame: Up to 45 days
Incremental recovery (K)
Time frame: Up to 45 days
Factor IX protein (FIX) activity
Time frame: Up to 45 days
Recombinant (FIXFc) concentration over time curves
Time frame: up to 45 days
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 19 Dec 2020.
- Derived from this study: PubMed 22110246Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallen B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood. 2012 Jan 19;119(3):666-72. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22.
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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.
Medical Director study director
Bioverativ Therapeutics Inc.
6 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 rFIXFc in people with Hemophilia B, run by Bioverativ Therapeutics Inc. at 6 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 8 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 19 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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?
Number of Participants experiencing Adverse Events (time frame: Up to 45 days). 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 NCT00716716, 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 19 Dec 2020.