by Ann Marie M. Minichiello

Published: Lifelines for Health Spring 2018

As research and medical advances progress, it is important to recognize that this momentum is not possible without the involvement of patients. Without clinical trials, new drugs and treatment options cannot be made available to those who would benefit from them. Presently, there are six clinical trials recruiting patients with hemophilia and inhibitors. The following studies are all “interventional” (clinical trials) with the primary purpose of treatment. The information on each of the six trials begins with its official title and drug being used.

1. Individualized ITI Based on Fviii(ATE) Protection by VWF (INITIATE) with Wilate® began on June 1, 2017 and is estimated to be completed by December 1, 2022. Requiring 120 participants, the primary goal of this trial is to compare the outcome of individualized lot selection to random lot selection using Wilate® for immune tolerance induction (ITI). The principal investigation site for this study is the University of California, Davis. (Sponsor: UC Davis)

2. A Non-controlled, Open-label, Multicenter,Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment , utilizing Biological: rFVIIIFc, began June 30, 2017 and is estimated to conclude December 2020. There are 30 participants needed for this study to describe the time to tolerization using rFVIIIFc within 12 months of ITI. There are 9 sites recruiting. (Sponsor: Bioverativ Therapeutics, Inc.)

3. A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2, using Biological: Coagulation FVIIa (Recombinant), began in December 2015 and was estimated to conclude August 30, 2017. Requiring 24 participants, the goal of this study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes. The principal investigation site is the University of Colorado, Denver, with 2 additional sites. (Sponsor: LFB USA, Inc.)

4. ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, With Inhibitory Antibodies to Factor VIII or IX is utilizing Fitusiran, recombinant Factor VIIa and FEIBA. This study began January 8, 2018 and is estimated to

   be completed in July 2019. Requiring 54 participants, the goal is to determine the frequency of bleeding episodes in patients receiving Fitusiran for prophylaxis, compared with patients who continue with their regular medication. There are 5 sites recruiting. (Sponsor: Alnylam Pharmaceuticals)

5. A Phase IV, Multicenter, Single-Arm, Open-Label Study of Emicizumab (Hemlibra) Prophylaxis in Patients With Hemophilia A and Inhibitors Undergoing Minor Surgical Procedures with Emicizumab began March 31, 2018 and is estimated to conclude June 28, 2019. 30 participants are needed for this study to evaluate whether participants receiving Emicizumab prophylaxis can safely undergo minor surgical procedures without prophylactic bypassing agents. There are 3 sites presently recruiting. (Sponsor: Genentech, Inc.)

6. A Phase III, Multicenter, Open-label Study of the Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (BAX 802) in Subjects With Congenital Hemophilia A With Factor VIII Inhibitors Undergoing Surgical or Other Invasive Procedures with Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802), began December 22, 2016 with an estimated completion date of June 1, 2018. There are 15 participants needed for this study to evaluate the efficacy and safety of BAX 802 in patients who are undergoing surgical or other invasive procedures. There is 1 site recruiting in the U.S. (Sponsor: Baxalta, part of Shire)

The safety and validity of a study is the responsibility of the sponsor and investigators. As with any clinical trial, please consult your healthcare provider (HTC) before participating. For more information, please visit www.clinicaltrials.gov.