Hemophilia BPhase 3NCT01271868
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
A Phase 3 study testing IB1001 in people with Hemophilia B, run by Medexus Pharma, Inc. at 10 sites in 3 countries.
Trial summary
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B. Read more about this study
- Who is it for?
- Ages Up to 12 years
- 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
- 9 participants
- Where
- 10 sitesin 3 countries
- Study dates
- Nov 2010to Dec 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
- Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
- Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
- Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
- 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
Show all 11Show fewer
- 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
- Willingness to participate in the trial for approximately 6 months (50 exposures)
- Age ≤12 years
- Hemoglobin ≥7 g/dL at the time of the blood draw
Who cannot join
- History of factor IX inhibitor ≥0.6 Bethesda units (BU)
- 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
- On medications that could impact hemostasis, such as aspirin
- 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
10 sites in 3 countries.
10 sites in 3 countries
India6 sites
AMRI Hospital, Institute of Haematology & Transfusion Medicine
Kolkata, 700073
Christian Medical College and Hospital
Ludhiana, 141008
Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
Pune, 411001
Sahyadri Speciality Hospital
Pune, 411004
Bhailal Amin General Hospital
Vadodara, 390003
Christian Medical College
Vellore, 632004
United States3 sites
RUSH University Medical Center
Chicago, Illinois, 60612
Gulf States Hemophilia & Thrombophilia Center
Houston, Texas, 77030
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
United Kingdom1 site
Great Ormond Street Hospital for Children
London, WC1N 3JH
About this study
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B.
- Conditions
- Hemophilia B
- Being tested
- IB1001
- Keywords
- Recombinant Factor IX
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IB1001Biological
Also called Recombinant factor IX, IXINITY
| 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 Infusions Required for Bleed Control
Measured: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months
Secondary outcomes (7)
Area Under the Curve (0-inf)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Terminal Half-life
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Concentration (Max)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Incremental Recovery
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Clearance
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Volume of Distribution (Steady State)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Measured: Pre-infusion to 72 hours following infusion
Annualized Bleed Rate
Measure was assessed during the Treatment Study
Measured: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2011 (estimated)
- Study startNov 2010
- Primary completionDec 2016
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medexus Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01271868
- Sponsor's protocol ID
- IB1001-02
- Status last verified
- Mar 2021
- First submitted
- 6 Jan 2011
- Last updated
- 15 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
- ClinicalTrials.gov ID
- NCT01271868
- Acronym / sponsor ID
- IB1001-02
- Conditions
- Hemophilia B
- Keywords
- Recombinant Factor IX
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Medexus Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2010 (actual)
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 7 Jan 2011 (estimated)
- Results
- Posted 11 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B.
Study design
- Primary purpose
- Treatment
- 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
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
- Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
- Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
- Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
- Platelet count at least 150,000/mm3
Key exclusion criteria (7 listed)
- History of factor IX inhibitor ≥0.6 Bethesda units (BU)
- 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
- On medications that could impact hemostasis, such as aspirin
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 |
|---|---|---|
| IB1001Also: Recombinant factor IX, IXINITY | Biological | IB1001 |
Primary outcome
Number of Infusions Required for Bleed Control
Time frame: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months
7 secondary outcomes
Area Under the Curve (0-inf)
Time frame: Pre-infusion to 72 hours following infusion
Terminal Half-life
Time frame: Pre-infusion to 72 hours following infusion
Concentration (Max)
Time frame: Pre-infusion to 72 hours following infusion
Incremental Recovery
Time frame: Pre-infusion to 72 hours following infusion
Clearance
Time frame: Pre-infusion to 72 hours following infusion
Volume of Distribution (Steady State)
Time frame: Pre-infusion to 72 hours following infusion
Annualized Bleed Rate
Time frame: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 3 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 Medexus Pharma, Inc. at 10 sites in 3 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 11 inclusion and 7 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: stopped early, as of the sponsor's last update on 15 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in India, 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?
Yes. Results are posted in the registry (first posted 11 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Infusions Required for Bleed Control (time frame: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 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 15 Mar 2021.