Phase 3 N=32 Randomized Quadruple-blind Supportive Care

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Hemophilia B

Bottom Line

View on ClinicalTrials.gov: NCT00768287 ↗

Enrolled (actual)

Serious AEs

13.0%

Results posted

Sep 2018

Primary outcome: Primary: Degree of Hemorrhage Control by Treatment Regimen — 53; 116; 105; 74 Bleeding Episodes

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: IB1001 (Biological); nonacog alfa (Biological)
Age: Pediatric, Adult, Older Adult · 5+ yrs
Sex: All
Sponsor: Medexus Pharma, Inc.
Primary completion: Mar 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY Degree of Hemorrhage Control by Treatment Regimen	53; 116; 105; 74; 25; 28	—
SECONDARY Area Under the Curve (0-inf)	1572.5; 1656.5; 1530.0	—
SECONDARY Area Under the Curve (0-72 hr)	1374.6; 1414.5; 1355.9	—
SECONDARY Terminal Half-life	24.2; 26.4; 24.2	—
SECONDARY Concentration (Max)	73.7; 72.8; 72.7	—
SECONDARY Incremental Recovery	0.98; 0.94; 0.94	—
SECONDARY Mean Residence Time	31.9; 35.4; 30.5	—
SECONDARY Clearance	0.051; 0.050; 0.052	—
SECONDARY Volume of Distribution (Steady State)	1.75; 1.81; 1.85	—
SECONDARY Annualized Bleed Rate	16.10; 1.52	—

Summary

Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to control of hemorrhaging in both the prophylaxis and on demand groups of IB1001 in subjects with hemophilia B. Key Secondary Objectives: To evaluate the ability of IB1001 to provide coverage against bleeding under surgical circumstances; To evaluate the long-term safety and efficacy of IB1001

Eligibility Criteria

Inclusion Criteria

Patient must be willing to give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent, make the required study visits, and follow instructions while enrolled in the study
Severe (factor IX activity ≤2 U/dL) hemophilia B subjects on demand therapy with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern as 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 150 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
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 up to 12-15 months
European Union (EU), Israel, and Canada: Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study [the Surgical Sub-study does not apply to the UK]; age of at least 12 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study

United States (US): Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study; age of at least 5 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study

Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00768287). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.