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

• ClinicalTrials.gov Identifier: NCT01125813
• Recruitment Status: Completed
• First Posted: May 18, 2010
• Results First Posted: April 22, 2013
• Last Update Posted: September 11, 2017
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Prevention
• Condition: Severe Hemophilia A
• Intervention: Biological: recombinant Factor VIII
• Enrollment: 32

Participant Flow

Recruitment Details	Participants were enrolled at 11 European sites beginning June 2010 and completing in January 2012.
Pre-assignment Details	36 participants were enrolled and screened. 4 were screen failures. Therefore, 32 of the 36 enrolled were exposed to investigation product.

Arm/Group Title	Human Cl-rhFVIII
Arm/Group Description	recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Period Title: Overall Study
Started	36
Completed	30
Not Completed	6
Reason Not Completed
Death	1
Withdrawal by Subject	1
Screen Failures	4

Baseline Characteristics

Arm/Group Title		Human Cl-rhFVIII
Arm/Group Description		recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Overall Number of Baseline Participants		32
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	32 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	31 96.9%
	>=65 years	1 3.1%
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	32 participants
		37.3 (13.6)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	32 participants
	Female	0 0.0%
	Male	32 100.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	32 participants
Austria		1
Bulgaria		8
Germany		8
United Kingdom		15

Outcome Measures

1. Primary Outcome

Title	Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months
Description	Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
Time Frame	At least 50 Exposure Days and at least 6 months

Outcome Measure Data

Analysis Population Description

All subjects who started prophylactic treatment were evaluated.

Arm/Group Title	Human Cl-rhFVIII
Arm/Group Description:	recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Overall Number of Participants Analyzed	32
Mean (Standard Deviation); Unit of Measure: Bleeds per month
	0.188 (0.307)

2. Primary Outcome

Title	Efficacy of Treating Bleeding Episodes
Description	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
Time Frame	After each bleeding episode, up to 6 month

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Human Cl-rhFVIII
Arm/Group Description:	recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
Overall Number of Participants Analyzed	32
Overall Number of Units Analyzed; Type of Units Analyzed: Bleeding episodes	28
Measure Type: Number; Unit of Measure: Percentage of treatments
Excellent	71.4
Good	28.6

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Human Cl-rhFVIII
Arm/Group Description	recombinant Factor VIII : intravenous infusion of factor FVIII every other day.
All-Cause Mortality
	Human Cl-rhFVIII
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	Human Cl-rhFVIII
	Affected / at Risk (%)
Total	2/32 (6.25%)
Injury, poisoning and procedural complications
Traumatic fracture	1/32 (3.13%)
Nervous system disorders
STATUS EPILEPTICUS	1/32 (3.13%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Human Cl-rhFVIII
	Affected / at Risk (%)
Total	3/32 (9.38%)
Gastrointestinal disorders
Abdominal pain	2/32 (6.25%)
Diarrhoea †	2/32 (6.25%)
Nausea †	2/32 (6.25%)
General disorders
Pyrexia	2/32 (6.25%)
Infections and infestations
Nasopharyngitis †	3/32 (9.38%)
Injury, poisoning and procedural complications
Contusion	2/32 (6.25%)
Musculoskeletal and connective tissue disorders
Back pain	2/32 (6.25%)
Nervous system disorders
Headache †	3/32 (9.38%)
Paraesthesia	2/32 (6.25%)
Respiratory, thoracic and mediastinal disorders
Epistaxis	2/32 (6.25%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Dr Sigurd Knaub
• Organization: Octapharma AG
• Phone: 41 55 451 21 41
• EMail: sigurd.knaub@octapharma.ch

• Responsible Party: Octapharma
• ClinicalTrials.gov Identifier: NCT01125813
• Other Study ID Numbers: GENA-08
• First Submitted: May 17, 2010
• First Posted: May 18, 2010
• Results First Submitted: January 21, 2013
• Results First Posted: April 22, 2013
• Last Update Posted: September 11, 2017