Trial Outcomes & Findings for Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX (NCT NCT00749476)

Results Overview

Clinical efficacy was measured by number/location of bleeding episodes, number of injections per bleeding, factor IX consumption, global assessment of efficacy by investigator and patient; biological efficacy (recovery) with BeneFIX was measured just after conversion.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

1 participants

Primary outcome timeframe

4 months

Results posted on

2011-06-08

Participant Flow

Patients were recruited in France from May 2008 to January 2009.

Before enrollment there was up to a 14 day screening period and the investigator reviewed the subject's medical history and medications to ensure that the subject was in good health and met all of the inclusion criteria and none of the exclusion criteria.

Participant milestones

Participant milestones
Measure	BeneFIX Plasma-derived FIX recovery with a dose of 50 ± 5 IU/kg before the conversion, BeneFIX recovery with a dose of 50 ± 5 IU/kg after the conversion, treatment with BeneFIX during the next 3 months.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BeneFIX n=1 Participants Plasma-derived FIX recovery with a dose of 50 ± 5 IU/kg before the conversion, BeneFIX recovery with a dose of 50 ± 5 IU/kg after the conversion, treatment with BeneFIX during the next 3 months.
Age Continuous	27.0 years STANDARD_DEVIATION 0.0 • n=93 Participants
Sex: Female, Male Female	0 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants

PRIMARY outcome

Timeframe: 4 months

Population: Intent to Treat (ITT) population. Due to low number of subjects that completed, no descriptive statistics for the efficacy endpoints are provided.

Clinical efficacy was measured by number/location of bleeding episodes, number of injections per bleeding, factor IX consumption, global assessment of efficacy by investigator and patient; biological efficacy (recovery) with BeneFIX was measured just after conversion.

Outcome measures

Outcome data not reported

Adverse Events

BeneFIX

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BeneFIX n=1 participants at risk Plasma-derived FIX recovery with a dose of 50 ± 5 IU/kg before the conversion, BeneFIX recovery with a dose of 50 ± 5 IU/kg after the conversion, treatment with BeneFIX during the next 3 months.
Infections and infestations Herpes simplex type-2 (HSV-2) meningitis	100.0% 1/1

Other adverse events

Other adverse events
Measure	BeneFIX n=1 participants at risk Plasma-derived FIX recovery with a dose of 50 ± 5 IU/kg before the conversion, BeneFIX recovery with a dose of 50 ± 5 IU/kg after the conversion, treatment with BeneFIX during the next 3 months.
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	100.0% 1/1
Gastrointestinal disorders Diarrhea	100.0% 1/1
Renal and urinary disorders Acute renal failure	100.0% 1/1
Musculoskeletal and connective tissue disorders Lower back pain	100.0% 1/1

Additional Information

U. S. Contact Center

Wyeth

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The PIs agreed to allow the sponsor 60 days to review and require changes to presentations or publications but only to protect confidential information and intellectual property, and for the sponsor to file a patent application, as applicable. The PIs also agreed for data to be presented first as a joint, multi-center publication.
Publication restrictions are in place

Restriction type: OTHER