Phase 3 N=120 Treatment

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B

Severe Hemophilia B

Bottom Line

View on ClinicalTrials.gov: NCT01425723 ↗

Enrolled (actual)

120

Serious AEs

28.5%

Results posted

Nov 2018

Primary outcome: Primary: Number of Participants With Any Positive Inhibitor Development — 0; 0; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: rFIXFc (Biological)
Age: Pediatric, Adult, Older Adult
Sex: Male
Sponsor: Bioverativ Therapeutics Inc.
Primary completion: Oct 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Any Positive Inhibitor Development	0; 0; 0	—
SECONDARY Annualized Bleeding Rate (ABR)	1.04; 1.14; 2.26; 3.69; 1.85; 0.54	—
SECONDARY Annualized Spontaneous Joint Bleeding Episodes	0.00; 0.00; 0.38; 0.00; 0.38; 0.00	—
SECONDARY Total Number of Exposure Days (EDs)	55.00; 165.00; 169.00; 54.00; 110.00; 157.00	—
SECONDARY Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])	3382.5; 3212.0; 2598.0; 3700.7; 2894.8; 3331.7	—
SECONDARY Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale	131; 622; 19; 184; 1; 9	—
SECONDARY Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale	60; 47; 342; 25; 336; 0	—

Summary

The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B. The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

Eligibility Criteria

Key Inclusion Criteria

Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Key Exclusion Criteria

High-titer inhibitor (>/=5.00 BU/mL)

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01425723). 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.