HOBOKEN, N.J.--(BUSINESS WIRE)--Octapharma
USA has announced that WILATE®,
von Willebrand Factor/Coagulation Factor VIII Complex (Human)
Lyophilized Powder for Solution for Intravenous Injection will be
utilized for two important clinical trials for hemophilia A launching in
“Individualized ITI Based on FVIII Protection by VWF
Octapharma USA will fund an investigator-initiated multi-center clinical
trial that aims to investigate one of the major challenges facing
hemophilia A treatment – the time it takes to decrease inhibitor levels.
Jonathan Ducore, M.D., M.P.H. of the University of California Davis
School of Medicine and Courtney Thornburg, M.D., M.S., of the Rady
Children's Hospital-San Diego are the principal investigators for the
study, entitled “Individualized ITI Based on FVIII Protection by VWF
(INITIATE).” The study evaluates a personalized medicine approach to
eradicating inhibitors in hemophilia A treatment.
The rationale is that an optimal regimen for immune tolerance induction
(ITI) has not been defined, and patients with hemophilia A and
inhibitors have high morbidity, mortality, and associated costs. In one
arm of the study, patients’ plasma will be tested against different WILATE®
lots in vitro before treatment to determine the batch that has the
highest residual Factor VIII activity left after incubation with the
“Anti-FVIII inhibitor formation is the most serious complication of
hemophilia A treatment today, occurring in up to 20 to 30 percent, or
more, of those with severe hemophilia A,” said Dr. Ducore. “Use of FVIII
is generally futile in this group, because the inhibitor will rapidly
inactivate the infused concentrate. ITI can result in inhibitor
resolution, but can take several years to achieve success. Preliminary
studies have suggested that individualized lot selection
(batch-matching) can shorten this time by 67 percent.”
This double-blinded, randomized clinical trial will compare the time to
inhibitor titer <0.6 BU using individualized lot selection and random
lot selection with the plasma-derived von Willebrand factor
(VWF)/coagulation factor (FVIII) complex concentrate for ITI in patients
with congenital hemophilia A, FVIII activity =2%, and a historical
high-titer inhibitor [=5 Bethesda Unit (BU)]. Participants will be
randomized on a one-to-one basis between the two study arms,
individualized lot selection (alternative treatment arm) and random lot
selection (standard treatment arm, current U.S. clinical practice in
ITI). Study sites, participants and investigators will be blinded to the
treatment status assigned. Octapharma will make the individually
selected factor lots available for subjects until the specific lot is
The time to inhibitor titer <0.6 BU, the study’s primary endpoint, is
hypothesized to correlate with a reduction in break-through bleeding,
morbidity, healthcare costs and improved quality of life. The Centers
for Disease Control and Prevention (CDC) recently demonstrated a
higher rate of mortality in FVIII inhibitor patients.1 In
multivariate analysis, the odds of death were 70 percent higher among
patients with a current inhibitor compared to those without an inhibitor
(p<0.01), and the deaths among inhibitor patients were primarily
attributed to bleeding complications.
Study participants are sought from U.S. hemophilia treatment centers
with congenital hemophilia with FVIII activity =2% and historical
high-inhibitor (=5 BU) will be eligible. The investigators plan to
enroll 120 participants, 60 in each arm of the study, who will meet
specific inclusion criteria.
For more information on the INITIATE clinical trial, please contact Dr.
Ducore at (916) 734-2781 or firstname.lastname@example.org;
or Dr. Thornburg at (858) 966-5811 or email@example.com.
The second study announced by Octapharma will investigate the
pharmacokinetics, efficacy, safety, and immunogenicity of WILATE®
in previously treated patients with severe hemophilia A. This
prospective, international, multi-center Phase 3 study seeks 55 male
participants at clinical trial sites in the U.S. and Europe.
will start in December. Study participants must have severe hemophilia A
and be age 12 or older. For more information, please contact Sylvia
Werner, Octapharma USA Director of Clinical Operations, at (201)
604-1149 or firstname.lastname@example.org.
The complete study protocol is available at www.ClinicalTrials.gov;
ClinicalTrials.gov Identifier: NCT02954575.
In the U.S., WILATE®
is indicated in children and adults with von Willebrand disease for
on-demand treatment and control of bleeding episodes; and perioperative
management of bleeding. WILATE® is not indicated for the
treatment of Hemophilia A in the U.S. Internationally, WILATE® is
approved for treatment in 62 countries.
Important Safety Information
is contraindicated for patients who have known hypersensitivity
reactions, including anaphylactic or severe systemic reaction, to human
plasma-derived products, any ingredient in the formulation, or
components of the container. Thromboembolic events have been reported in
VWD patients receiving coagulation factor replacement therapies. FVIII
activity should be monitored to avoid sustained excessive FVIII levels.
Development of neutralizing antibodies to FVIII and to VWF, especially
in VWD Type 3 patients, may occur. WILATE® is made from human
plasma. The risk of infectious agents, including viruses and,
theoretically, the Creutzfeldt-Jacob disease agent, cannot be completely
eliminated. The most common adverse reactions to treatment with WILATE®
in patients with VWD were hypersensitivity reactions, urticaria, and
dizziness. Seroconversions for antibodies to parvovirus B19 not
accompanied by clinical signs of disease have been observed. The most
serious adverse reactions to treatment with WILATE® in
patients with VWD were hypersensitivity reactions. For full prescribing
information on WILATE®,
please visit www.wilateusa.com.
About the Octapharma Group
Headquartered in Lachen, Switzerland, Octapharma
is one of the largest human protein products manufacturers in the world
and has been committed to patient care and medical innovation since
1983. Its core business is the development and production of human
proteins from human plasma and human cell lines. Octapharma employs
approximately 6,000 people worldwide to support the treatment of
patients in over 100 countries with products across the following
therapeutic areas: Hematology (coagulation disorders), Immunotherapy
(immune disorders) and Critical Care. The company’s American subsidiary,
Octapharma USA, is located in Hoboken, N.J. Octapharma operates two
state-of-the-art production sites licensed by the U.S.
Food and Drug Administration (FDA), providing a high level of
production flexibility. For more information, please visit www.octapharmausa.com.
1 - Walsh CE, Soucie JM, Miller CH, the United States Hemophilia
Treatment Center N. Impact of inhibitors on hemophilia a mortality in
the United States. American Journal of Hematology. Jan 23 2015.