The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will investigate the ability of Eloctate (efmoroctocog alfa), a recombinant clotting factor therapy developed for hemophilia A, to induce immune tolerance, a process scientists describe as immune tolerance induction (ITI) potential, for up to 60 weeks.
The development of an inhibitor is a major complication faced by patients with hemophilia A who have been treated with factor VIII (FVIII) therapies. In these patients, the body produces inhibitors (antibodies) directed against the foreign factor proteins. This renders standard replacement therapy ineffective and increases the risk of severe bleeding and morbidity, which also results in soaring healthcare costs.
Previous Phase 3 clinical trials — A-LONG (NCT01181128), Kids ALONG (NCT01458106), and the ongoing ASPIRE extension study (NCT01454739) — showed Eloctate’s long-term effectiveness as a therapy for acute bleeding episodes. Additionally, Eloctate showed effectiveness in surgical and preventive management in previously treated males with severe hemophilia A.
The primary objective of the ReITIrate clinical trial is to evaluate the number of patients who achieve ITI success, determined by the following three criteria: being negative for inhibitor (according to the Nijmegen-modified Bethesda assay) at two consecutive visits; showing an increase in factor VIII incremental recovery (IR) of at least 66% of the expected IR at two consecutive visits; and a factor half-life above seven hours.
Participants will be treated with Eloctate at 200 international units (IU)/kilogram (kg), administered once daily in an injection, or in several injections throughout the day. The study will continue for a maximum of 60 weeks.
Participants who meet the criteria for ITI success will receive tapered doses over 16 weeks until a prophylactic, or preventive, dose is achieved as defined by the investigator. Then, participants will be followed for 32 weeks while receiving prophylactic treatment with Eloctate. The study’s estimated completion date is April 2020.
“We are committed to provide treatment options to patients with haemophilia that will enable them to make choices and live the lives they would like to live. The ReITIrate study is very much aligned with that commitment and may address one of the most critical questions – treatment management of patients who have developed inhibitors,” Krassimir Mitchev, MD, PhD, vice president and medical therapeutic area head of hemophilia at Sobi, said in a press release.
Eloctate is approved and marketed in the United States by Bioverativ Therapeutics for the treatment of hemophilia A. In Europe, the therapy is sold under the name Elocta and marketed by Sobi.