Clinical trial NCT02234310: Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
Official title: An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia B
Keywords: prophylaxis treatment
episodic treatment
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Swedish Orphan Biovitrum
First posted: 2014-09-09
Enrollment: 33
Sex: MALE
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.


Eligibility criteria: Key Inclusion Criteria:

* Weight \>=3.5 kilogram at the time of informed consent.
* Severe hemophilia B was defined as less than or equal to (\<=)2 International Units per deciliter (IU/dL) (\<=2 percent \[%\]) endogenous FIX documented in the medical record or as tested during the Screening Period.

Key Exclusion Criteria:

* History of positive inhibitor testing. A prior history of inhibitors was defined based on a participant's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (that is equal to or above lower limit of detection).
* History of hypersensitivity reactions associated with any rFIXFc administration.
* Exposure to blood components or injection with a coagulation factor IX (FIX) concentrate (including plasma derived) other than rFIXFc.
* Injection with commercially available rFIXFc more than 28 days prior to Screening.
* More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility.
* Other coagulation disorders in addition to hemophilia B.
* Any concurrent clinically significant major disease that, in the opinion of the Investigator, would have made the participant unsuitable for enrollment (example HIV infection with cluster of differentiation 4 (CD4) lymphocyte count less than (\<)200 cells/microliter (mcL) or a viral load greater than (\>)200 particles/mcL, or any other known congenital or acquired immunodeficiency).
* Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes was allowed. Treatment in these circumstances should not have exceeded a 14-day duration.
* Participation within the past 30 days in any other clinical study involving investigational treatment.
* Current enrollment in any other clinical study involving investigational treatment.
* Inability to comply with study requirements.
* Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, would have made the participant unsuitable for enrollment.

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
timeFrame: Up to 3 years
Secondary outcomes:
measure: Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
timeFrame: Up to 3 years
measure: Annualized Number of Spontaneous Joint Bleeding Episodes
timeFrame: Up to 3 years
measure: Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
timeFrame: Up to 3 years
measure: Total Number of Exposure Days (EDs)
timeFrame: Up to 3 years
measure: Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
timeFrame: Up to 3 years
measure: Number of Injections of rFIXFc Required to Resolve a Bleeding Episode
timeFrame: Up to 3 years
measure: Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode
timeFrame: Up to 3 years
measure: Change From Baseline in rFIXFc Incremental Recovery (IR)
timeFrame: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: Research Site
state: California
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: Louisville
country: United States
facility: Research Site
state: Kentucky
city: New Orleans
country: United States
facility: Research Site
state: Louisiana
city: East Lansing
country: United States
facility: Research Site
state: Michigan
city: Traverse City
country: United States
facility: Research Site
state: Michigan
city: Columbus
country: United States
facility: Research Site
state: Ohio
city: Portland
country: United States
facility: Research Site
state: Oregon
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Westmead
country: Australia
facility: Research Site
state: New South Wales
city: Aarhus
country: Denmark
facility: Research Site
city: Lille
country: France
facility: Hopital Cardiologique - CHU Lille
state: Nord
city: Lyon Cedex 3
country: France
facility: Research Site
state: Rhone
city: Dublin
country: Ireland
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Parma
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Utrecht
country: Netherlands
facility: Research Site
city: Auckland
country: New Zealand
facility: Research Site
city: Warszawa
country: Poland
facility: Research Site
city: Malmo
country: Sweden
facility: Research Site
city: Cambridge
country: United Kingdom
facility: Research Site
state: Cambridgeshire
city: Whitechapel
country: United Kingdom
facility: Research Site
state: London
city: London
country: United Kingdom
facility: Research Site
Study officials:
References:
citation: Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-2739. doi: 10.1182/bloodadvances.2020004085.