Hemophilia BPhase 3NCT02048111
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
A Phase 3 study testing IB1001 in people with Hemophilia B, run by Cangene Corporation at 2 sites in 2 countries.
Trial summary
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia B
- What is being tested?
- IB1001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Mar 2014to Nov 2015
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age of at least 12 years
- Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
- Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
- Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
- Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis
- Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study
Show all 14Show fewer
- Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
- Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations
- Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
- Platelet count at least 150,000/mm3
- Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times the upper limit of the normal range
- Total bilirubin ≤1.5 times the upper limit of the normal range
- Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
- Hemoglobin ≥7 g/dL at the time of the blood draw
Who cannot join
- History of factor IX inhibitor ≥0.6 BU (Bethesda units)
- Existence of another coagulation disorder
- Evidence of thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
- Use of an investigational drug within 30 days prior to study entry
- Previous use of IB1001
- Use of medications that could impact hemostasis, such as aspirin
Show all 10Show fewer
- Hypersensitivity to the active substance or to any of the excipients in the investigational products
- Known allergic reaction to hamster proteins
- History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol
- History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.
Read the detailed description
Primary Objectives:
- to evaluate safety of IB1001 within the first 50 exposure days,
- to determine IB1001 pharmacokinetics (PK), and
- to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days
Secondary Objectives:
- to evaluate long-term safety of IB1001; and
- to evaluate long term efficacy of IB1001.
Exploratory Objectives:
- to evaluate markers of thrombogenicity during the first 24 hours post-infusion [thrombogenicity markers will include at a minimum D-dimer test; however should there be a clinical reason (e.g., three consecutive elevations in D-dimer levels, a possible clinical thrombogenic episode), sufficient samples will be collected to also evaluate levels of fragment 1+2 (F1+2) and thrombin-antithrombin III complex (TAT)]
- to evaluate IB1001 immunogenicity response (development of inhibitory and non-inhibitory factor IX binding antibodies and antibodies to host cell proteins)
- Conditions
- Hemophilia B
- Being tested
- IB1001
- Keywords
- Hemophilia B, Hemophilia A, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked
- Medical subject headings
- Hemophilia B, Hemophilia A, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IB1001Biological
Prophylaxis (during Treatment and Continuation phases): 40 - 75 IU/kg twice weekly.
The starting dose for prophylaxis may be based on previous recombinant factor IX product use. The recommended starting prophylaxis dose is 40 - 60 IU/kg twice weekly, however, the investigator may prescribe up to 75 IU/kg twice weekly at their discretion (after clinically assessing the subject) and discretion of the subject.
The dose or the frequency of IB1001 prophylaxis may be adjusted at the discretion of the investigator.
| Study group | Role | What they receive |
|---|---|---|
| IB1001 | Experimental | Not described Receives: Biological: IB1001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of study subjects with adverse events
Information on adverse events is collected after each infusion of study drug by a study subject. Assessment of adverse events is then performed by an investigator after 5 infusions of study drug, 1 month, 2 months, 3 months and 6 months of study drug treatment.
Measured: Within 6 months
Secondary outcomes (1)
Number of bleeding episodes divided by number of months of observation
Measured: Within 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2014 (estimated)
- Study startMar 2014
- Primary completionOct 2015 (estimated)
- Study completionNov 2015 (estimated)
Why it stopped: The study was withdrawn from the IND.
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 3
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cangene Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02048111
- Sponsor's protocol ID
- IB1001-04
- Status last verified
- Aug 2016
- First submitted
- 14 Nov 2013
- Last updated
- 23 Aug 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B
- ClinicalTrials.gov ID
- NCT02048111
- Acronym / sponsor ID
- IB1001-04
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, Hemophilia A, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study was withdrawn from the IND.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Cangene CorporationIndustry (pharma, biotech, devices)
- Study start
- Mar 2014
- Primary completion
- Oct 2015 (estimated)
- Study completion
- Nov 2015 (estimated)
- First posted
- 29 Jan 2014 (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.
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.
Detailed description
Primary Objectives:
- to evaluate safety of IB1001 within the first 50 exposure days,
- to determine IB1001 pharmacokinetics (PK), and
- to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days
Secondary Objectives:
- to evaluate long-term safety of IB1001; and
- to evaluate long term efficacy of IB1001.
Exploratory Objectives:
- to evaluate markers of thrombogenicity during the first 24 hours post-infusion [thrombogenicity markers will include at a minimum D-dimer test; however should there be a clinical reason (e.g., three consecutive elevations in D-dimer levels, a possible clinical thrombogenic episode), sufficient samples will be collected to also evaluate levels of fragment 1+2 (F1+2) and thrombin-antithrombin III complex (TAT)]
- to evaluate IB1001 immunogenicity response (development of inhibitory and non-inhibitory factor IX binding antibodies and antibodies to host cell proteins)
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IB1001 | Experimental | Biological: IB1001 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- Age of at least 12 years
- Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
- Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
- Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
- Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis
Key exclusion criteria (10 listed)
- History of factor IX inhibitor ≥0.6 BU (Bethesda units)
- Existence of another coagulation disorder
- Evidence of thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
- Use of an investigational drug within 30 days prior to study entry
- Previous use of IB1001
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 |
|---|---|---|
| IB1001Prophylaxis (during Treatment and Continuation phases): 40 - 75 IU/kg twice weekly. The starting dose for prophylaxis may be based on previous recombinant factor IX product use. The recommended starting prophylaxis dose is 40 - 60 IU/kg twice weekly, however, the investigator may prescribe up to 75 IU/kg twice weekly at their discretion (after clinically assessing the subject) and discretion of the subject. The dose or the frequency of IB1001 prophylaxis may be adjusted at the discretion of the investigator. | Biological | IB1001 |
Primary outcome
Number of study subjects with adverse events
Time frame: Within 6 months
Information on adverse events is collected after each infusion of study drug by a study subject. Assessment of adverse events is then performed by an investigator after 5 infusions of study drug, 1 month, 2 months, 3 months and 6 months of study drug treatment.
1 secondary outcome
Number of bleeding episodes divided by number of months of observation
Time frame: Within 12 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2016.
Related trials for Hemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IB1001 in people with Hemophilia B, run by Cangene Corporation at 2 sites in 2 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 14 inclusion and 10 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: withdrawn, as of the sponsor's last update on 23 Aug 2016. 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, United Kingdom. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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 study subjects with adverse events (time frame: Within 6 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 Aug 2016.