Trial Outcomes & Findings for Wilate in Subjects With Von Willebrand Disease Who Undergo Surgery (NCT NCT01365546)

Last Updated: 2015-03-09

Results Overview

Efficacy of Wilate in surgical procedures was assessed intra-operatively by the surgeon and post-operatively by the investigator. The IDMC additionally conducted an independent adjudication of all hemostatic efficacy results ('secondary adjudication') and adjudicated the surgeons'/investigators' assessments of the intra- and post-operative assessments where there were discrepancies between the two assessments ('primary adjudication'). It was specified in the SAP that the study will be terminated early and success claimed if the two-sided 98.75% confidence interval (CI) for the overall success rate excludes and is greater than 0.60 (equivalent to 25 or more successes out of the 30 procedures).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

30 participants

Primary outcome timeframe

30 Days

Results posted on

2015-03-09

Participant Flow

Participant milestones

Participant milestones
Measure	Human VWF/FVIII Concentrate human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery
Overall Study STARTED	41
Overall Study COMPLETED	30
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Human VWF/FVIII Concentrate human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery
Overall Study Withdrawal by Subject	1
Overall Study Screen failure	9
Overall Study Study termination	1

Baseline Characteristics

Wilate in Subjects With Von Willebrand Disease Who Undergo Surgery

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Human VWF/FVIII Concentrate n=41 Participants human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery
Age, Continuous	39.7 years STANDARD_DEVIATION 18.1 • n=5 Participants
Sex: Female, Male Female	29 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants
Region of Enrollment Poland	2 participants n=5 Participants
Region of Enrollment Oman	2 participants n=5 Participants
Region of Enrollment Romania	8 participants n=5 Participants
Region of Enrollment Turkey	1 participants n=5 Participants
Region of Enrollment Bulgaria	1 participants n=5 Participants
Region of Enrollment South Africa	2 participants n=5 Participants
Region of Enrollment Italy	5 participants n=5 Participants
Region of Enrollment India	13 participants n=5 Participants

PRIMARY outcome

Timeframe: 30 Days

Outcome measures

Outcome measures
Measure	Minor Surgery n=9 Participants human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery	Major Surgery n=21 Participants	All Surgeries n=30 Participants
Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale.	9 participants In this study a CI of 98.75% was used. CI: 0.569, 1.000. The success rate is 100%	20 participants In this study a CI of 98.75% was used. CI: 0.704, 1.000. The success rate is 95.2%	29 participants In this study a CI of 98.75% was used. CI: 0.784, 1.000. The success rate is96.7%

SECONDARY outcome

Timeframe: 1 Day

The efficacy of Wilate during surgical procedures was assessed by a 4-point ordinal efficacy scale by the surgeon at the end of the surgical procedure and took the predicted versus actual blood loss and transfusion requirements into consideration. Outcome measure 1 takes the results of outcome measure 2 and 3 into consideration and is an overall assessment covering intra- and post-operative efficacy.

Outcome measures

Outcome measures
Measure	Minor Surgery n=30 Participants human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery	Major Surgery	All Surgeries
Assessment of Intra-operative Hemostatic Efficacy	27 participants	—	—

SECONDARY outcome

Timeframe: up to 30 days

Post-operative efficacy was assessed by the investigator, covering the time period from the end of the procedure up to 24 hours following the last infusion of study medication. This assessment took the post-operative bleeding and oozing into consideration

Outcome measures

Outcome measures
Measure	Minor Surgery n=30 Participants human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery	Major Surgery	All Surgeries
Post-operative Efficacy Assessment	27 participants	—	—

Adverse Events

Human VWF/FVIII Concentrate

Serious events: 2 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Human VWF/FVIII Concentrate n=41 participants at risk human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery
Reproductive system and breast disorders Vaginal Hemorrhage	2.4% 1/41 • Number of events 1 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
Gastrointestinal disorders Gastritis erosive	2.4% 1/41 • Number of events 1 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.

Other adverse events

Other adverse events
Measure	Human VWF/FVIII Concentrate n=41 participants at risk human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery
Injury, poisoning and procedural complications Procedural Pain	19.5% 8/41 • Number of events 10 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
Gastrointestinal disorders Nausea	14.6% 6/41 • Number of events 7 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
Gastrointestinal disorders Vomiting	14.6% 6/41 • Number of events 6 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
General disorders Pain	9.8% 4/41 • Number of events 4 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
General disorders Pyrexia	9.8% 4/41 • Number of events 4 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
Investigations Hemoglobin decreased	9.8% 4/41 • Number of events 6 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.
Vascular disorders Hypertension	9.8% 4/41 • Number of events 4 • Adverse events were collected for each patient over a period of 1 to approximately 6 month from signing the informed consent to study completion.

Additional Information

Director of Clinical Operations

Octapharma

Phone: 4152609577

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.
Publication restrictions are in place

Restriction type: OTHER