NC-HEM-12-4: A Phase 3, Multicenter, Randomized, Double-blind,Active-controlled, Parallel-group Trial with an Open-label ExtensionPhase to Evaluate the Efficacy and Safety of Oral E5501 versusEltrombopag, in Adults with Chronic Immune Thrombocytopenia(Idiopathic Thrombocytopenic Purpura)
The study consisted of three phases: Prerandomization, Randomization (Core Study) and
Extension Phase. Participants 18 years of age and over, who meet all the eligibility
requirements, were randomized into the study. It was required that splenectomized
participants made up at least 35% of the study population and no single platelet count was
greater than 35x10^9/L. Participants were centrally stratified at randomization by
splenectomy status, baseline platelet count, and use of concomitant ITP medication at
baseline and were randomized to receive either double-blind avatrombopag or eltrombopag in a
1:1 ratio. Participants received blinded therapy at a starting dose of 20 mg avatrombopag
once daily or 50 mg eltrombopag once daily. Participants were allowed to have their dose
titrated up (maximum dose 40 mg avatrombopag and 75 mg for eltrombopag) or down (minimum dose
5 mg for avatrombopag and 25 mg for eltrombopag) depending on their response to study drug.
The goal of dose modification was to maintain the platelet count at levels greater than or
equal to 50x10^9/L and less than or equal to 150x10^9/L, and to decrease the need for
ITP-directed concomitant medications. The duration of treatment in the Core study and the
Extension Phase is approximately 26 and 104 weeks, respectively.
A Phase I/II Study of Immunotherapy with Humanized Anti-CD20
Antibody, IMMU-106 (hA20), in Adult Patients with Chronic Immune
Idiopathic thrombocytopenic purpura (ITP) is a condition in which the patient has very low platelet counts. It is believed that this is caused by the body's own immune system destroying the platelets. Initial treatment is with steroids and if this is not successful, removal of the spleen may be performed. Sometimes, the patient is refractory to all forms of therapy. CD20 is an antigen that is found on B cells. B cells are important in the immune system. Anti-CD20 is an antibody that targets the CD20 site on the B cell and can bind to this site inhibiting an important step in the proliferation of B cells thereby causing a depletion of these cells. The purpose of this study is to determine the optimal dose of the experimental agent IMMU-106 (an anti-CD-20 antibody) in patients with ITP. This is a Phase I/II study in which 3 different doses ( administered twice, 2 weeks apart, intravenously or by subcutaneous injection) will be evaluated for safety and to determine the optimal dose for later studies. An additional dosing schedule with hA20 administered by subcutaneous injection at a dose of 320 mg given once weekly for 4 consecutive weeks has been implemented. Initial subjects will be given infusions of the drug at the lowest of the three doses. If there is no significant toxicity, the next group of subjects will receive a higher dose and if there is again no significant toxicity, the third group will receive the highest dose. The subjects will be monitored for response to the drug, adverse effects, labs, physical exams, medical status and EKGs.