Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

INdividualized ITI Based on Fviii(ATE) Protection by VWF (INITIATE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03204539
Recruitment Status : Terminated (Termination of funding from sponsor.)
First Posted : July 2, 2017
Results First Posted : May 29, 2020
Last Update Posted : May 29, 2020
Sponsor:
Information provided by (Responsible Party):
University of California, Davis

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Hemophilia A With Inhibitor
Hemophilia A
Intervention Drug: Wilate
Enrollment 1
Recruitment Details Only 1 participant enrolled, but did not complete study due to early study termination.
Pre-assignment Details  
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Period Title: Overall Study
Started 0 1
Completed 0 0
Not Completed 0 1
Reason Not Completed
Loss of funding             0             1
Arm/Group Title Alternative Treatment Standard Treatment Total
Hide Arm/Group Description

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Total of all reporting groups
Overall Number of Baseline Participants 0 1 1
Hide Baseline Analysis Population Description
Only 1 participant enrolled (standard treatment arm), and did not complete study due to loss of funding.
Age, Customized  
Measure Type: Number
Unit of measure:  Years
Age Number Analyzed 0 participants 1 participants 1 participants
5 5
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 1 participants 1 participants
Female
0
   0.0%
0
   0.0%
Male
1
 100.0%
1
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 1 participants 1 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
Not Hispanic or Latino
1
 100.0%
1
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 1 participants 1 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
White
1
 100.0%
1
 100.0%
More than one race
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 0 participants 1 participants 1 participants
1 1
1.Primary Outcome
Title Time to Negative Inhibitor
Hide Description This endpoint was chosen because a shorter time to negative inhibitor should decrease monthly break-through bleeding frequency in the early phase of ITI
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Time to Achieve Partial and Complete Success
Hide Description

Secondary endpoints include time to achieve partial and complete success as defined according to the following criteria:

  • Inhibitor titer <0.6 BU.
  • Incremental in vivo FVIII recovery in the normal range [≥66% of normal (1.5% per IU/kg), equal to 0.99%per IU/kg] with samples taken prior to and 15 or 30 minutes after concentrate treatment. The recovery assessment should be done without any wash-out period.
  • Half-life of FVIII >6 hours. The half-life assessment should be done in a non-bleeding status without any wash-out period.

Complete Success (CS) of ITI:

All three criteria above met.

Partial Success (PS) of ITI:

The first two of the three criteria above met.

Partial Response (PR) of ITI:

One of the three criteria above met.

Partial Failure (PF) of ITI:

Inhibitor still present, but titer is decreased to <5 BU in contrast to ≥5 BU before start.

Complete Failure (CF) of ITI:

None of the above mentioned criteria met, and the inhibitor titer is still ≥5 BU.

Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Absence of Relapse, up to 12 Months After Achievement of Complete or Partial ITI Success
Hide Description [Not Specified]
Time Frame one year after completion of immune tolerance induction, up to 30 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title The Number of Break-through Bleeding Events During the Course of ITI-treatment·
Hide Description [Not Specified]
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Cost of ITI - Including Bleeding Control Using Bypassing Agents Prior to Start and During ITI
Hide Description [Not Specified]
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Subject Quality of Life
Hide Description measured with the Haemo-QOL questionnaire
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Subject Compliance With ITI Treatment Regimen
Hide Description We will be looking at drug accountability reports/ logs which will reflect each subject's usage of Wilate
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title The Impact of Inhibitor Titer at Start of ITI and During the Course of ITI, Including the Peak Titer of the Inhibitor
Hide Description [Not Specified]
Time Frame completion of immune tolerance induction, up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
9.Secondary Outcome
Title Understand Other Factors Related to ITI Success Using Additional Biologic Assays
Hide Description

If subject consents, the following assays will be performed:

epitope mapping immunogenotyping/HLA genotyping FVIII genetic testing

Time Frame screening/baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Study terminated, and no participants completed study. No outcome measure data to report.
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description:

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 6 months
Adverse Event Reporting Description Only 1 participant enrolled in study (Standard treatment arm) due to loss of funding.
 
Arm/Group Title Alternative Treatment Standard Treatment
Hide Arm/Group Description

Half of the participants will be randomized to blinded individualized lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

The other half of the participants will receive random lot selection for ITI.

Wilate: Wilate® is a high-purity (i.e. 100 IU FVIII/mg total protein) pdVWF/FVIII complex concentrate.Wilate® possesses all the important features asked for in ITI, namely high purity, a very high pathogen safety profile, and an excellent protection of its FVIII by VWF – all achieved through unique, novel, and innovative techniques.

All-Cause Mortality
Alternative Treatment Standard Treatment
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0      0/1 (0.00%)    
Hide Serious Adverse Events
Alternative Treatment Standard Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/0      1/1 (100.00%)    
Infections and infestations     
Sepsis *  0/0  0 1/1 (100.00%)  1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Alternative Treatment Standard Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/0      1/1 (100.00%)    
General disorders     
Headache *  0/0  0 1/1 (100.00%)  1
Infections and infestations     
Fever *  0/0  0 1/1 (100.00%)  1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Jonathan Ducore
Organization: UC Davis
Phone: 916-734-3461
EMail: jmducore@ucdavis.edu
Layout table for additonal information
Responsible Party: University of California, Davis
ClinicalTrials.gov Identifier: NCT03204539    
Other Study ID Numbers: 988465
First Submitted: June 13, 2017
First Posted: July 2, 2017
Results First Submitted: May 14, 2020
Results First Posted: May 29, 2020
Last Update Posted: May 29, 2020