Clinical Trials Logo

Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT03204539
Other study ID # 988465
Secondary ID
Status Terminated
Phase Phase 4
First received
Last updated
Start date June 1, 2017
Est. completion date July 19, 2019

Study information

Verified date May 2020
Source University of California, Davis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex concentrate for immune tolerance induction (ITI) in subjects with congenital Hemophilia A, FVIII activity ≤2%, and a historical high-titer inhibitor [≥5 Bethesda Unit (BU)].


Description:

Read more »
Read more »

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
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.

Locations

Country Name City State
United States Tulane University New Orleans Louisiana
United States University of California, Davis Sacramento California
United States Rady Children's Hospital San Diego San Diego California

Sponsors (1)

Lead Sponsor Collaborator
University of California, Davis

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Time to Negative Inhibitor This endpoint was chosen because a shorter time to negative inhibitor should decrease monthly break-through bleeding frequency in the early phase of ITI completion of immune tolerance induction, up to 18 months
Secondary Time to Achieve Partial and Complete Success 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.
completion of immune tolerance induction, up to 18 months
Secondary Absence of Relapse, up to 12 Months After Achievement of Complete or Partial ITI Success one year after completion of immune tolerance induction, up to 30 months
Secondary The Number of Break-through Bleeding Events During the Course of ITI-treatment· completion of immune tolerance induction, up to 18 months
Secondary Cost of ITI - Including Bleeding Control Using Bypassing Agents Prior to Start and During ITI completion of immune tolerance induction, up to 18 months
Secondary Subject Quality of Life measured with the Haemo-QOL questionnaire completion of immune tolerance induction, up to 18 months
Secondary Subject Compliance With ITI Treatment Regimen We will be looking at drug accountability reports/ logs which will reflect each subject's usage of Wilate completion of immune tolerance induction, up to 18 months
Secondary The Impact of Inhibitor Titer at Start of ITI and During the Course of ITI, Including the Peak Titer of the Inhibitor completion of immune tolerance induction, up to 18 months
Secondary Understand Other Factors Related to ITI Success Using Additional Biologic Assays If subject consents, the following assays will be performed:
epitope mapping immunogenotyping/HLA genotyping FVIII genetic testing
screening/baseline
See also
  Status Clinical Trial Phase
Completed NCT03834727 - Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
Completed NCT03191799 - A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Phase 3
Completed NCT01599819 - BAX 855 Dose-Escalation Safety Study Phase 1
Terminated NCT04541628 - Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A Phase 1/Phase 2
Completed NCT02847637 - A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors Phase 3
Completed NCT04072237 - Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia Phase 1
Completed NCT04085458 - Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) Phase 4
Completed NCT04565236 - A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A Phase 4
Recruiting NCT05987449 - A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A Phase 1/Phase 2
Active, not recruiting NCT04621916 - Preventing Inhibitor Recurrence Indefinitely Phase 4
Not yet recruiting NCT02888223 - Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A Phase 1
Completed NCT02528968 - National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A N/A
Completed NCT02225483 - Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function N/A
Completed NCT02199717 - An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia N/A
Completed NCT01217255 - Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Completed NCT00969319 - Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America N/A
Terminated NCT00995046 - Individually Tailored Prophylaxis in Patients With Severe Hemophilia A N/A
Completed NCT00868530 - Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects Phase 3
Completed NCT00839202 - Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay N/A
Completed NCT00629837 - Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980 Phase 1