Trial Outcomes & Findings for Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A (NCT NCT01125813)

NCT ID: NCT01125813

Last Updated: 2017-09-11

Results Overview

Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 32 participants
• Primary outcome timeframe: At least 50 Exposure Days and at least 6 months
• Results posted on: 2017-09-11

Participant Flow

Participants were enrolled at 11 European sites beginning June 2010 and completing in January 2012.

36 participants were enrolled and screened. 4 were screen failures. Therefore, 32 of the 36 enrolled were exposed to investigation product.

Participant milestones

Measure	Human Cl-rhFVIII recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Overall Study STARTED	36
Overall Study COMPLETED	30
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Measure	Human Cl-rhFVIII recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Overall Study Death	1
Overall Study Withdrawal by Subject	1
Overall Study Screen Failures	4

Baseline Characteristics

Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A

Baseline characteristics by cohort

Measure	Human Cl-rhFVIII n=32 Participants recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	31 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	37.3 years STANDARD_DEVIATION 13.6 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	32 Participants n=5 Participants
Region of Enrollment Austria	1 participants n=5 Participants
Region of Enrollment Bulgaria	8 participants n=5 Participants
Region of Enrollment Germany	8 participants n=5 Participants
Region of Enrollment United Kingdom	15 participants n=5 Participants

PRIMARY outcome

Timeframe: At least 50 Exposure Days and at least 6 months

Population: All subjects who started prophylactic treatment were evaluated.

Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.

Outcome measures

Measure	Human Cl-rhFVIII n=32 Participants recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months	0.188 Bleeds per month Standard Deviation 0.307

PRIMARY outcome

Timeframe: After each bleeding episode, up to 6 month

At the end of a bleeding episode, efficacy was assessed as:

• Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion
• Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution
• Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution
• None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated

Outcome measures

Measure	Human Cl-rhFVIII n=28 bleeding episodes recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Efficacy of Treating Bleeding Episodes Excellent	71.4 Percentage of treatments
Efficacy of Treating Bleeding Episodes Good	28.6 Percentage of treatments

Adverse Events

Human Cl-rhFVIII

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Human Cl-rhFVIII n=32 participants at risk recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Injury, poisoning and procedural complications Traumatic fracture	3.1% 1/32
Nervous system disorders STATUS EPILEPTICUS	3.1% 1/32

Other adverse events

Measure	Human Cl-rhFVIII n=32 participants at risk recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Infections and infestations Nasopharyngitis	9.4% 3/32
Nervous system disorders Headache	9.4% 3/32
Gastrointestinal disorders Abdominal pain	6.2% 2/32
Gastrointestinal disorders Diarrhoea	6.2% 2/32
Gastrointestinal disorders Nausea	6.2% 2/32
General disorders Pyrexia	6.2% 2/32
Injury, poisoning and procedural complications Contusion	6.2% 2/32
Musculoskeletal and connective tissue disorders Back pain	6.2% 2/32
Nervous system disorders Paraesthesia	6.2% 2/32
Respiratory, thoracic and mediastinal disorders Epistaxis	6.2% 2/32

Additional Information

Dr Sigurd Knaub

Octapharma AG

Phone: 41 55 451 21 41

Email: sigurd.knaub@octapharma.ch

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60