Purpura, Thrombotic Thrombocytopenic Clinical Trial
Official title:
A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
To evaluate the overall safety and tolerability of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with three types of von Willebrand Factor related platelet disorders.
ARC1779 Injection will be investigated in 4 cohorts of TTP patients as an uncontrolled,
open-label study. Patients with vWD-2b will be enrolled in an additional cohort in a
randomized, blinded, double-dummy, and placebo-controlled study.
Collectively, patients representing 3 different vWF-related platelet function disorders: TTP
in remission, acute TTP, and vWD-2b will be treated in a total of 5 cohorts. Three cohorts
will consist of patients who are status post an episode of TTP ("TTP Remission Cohorts") and
will be treated with ARC1779 Injection in a dose- and duration-escalation design. In
parallel, a single cohort of patients with acute TTP ("Acute TTP Cohort") will be treated
according to an individual patient titration-to-response paradigm. This cohort will be
opened for enrollment at the beginning of the study and closed after all of the other
cohorts are completed. Also in parallel, a single cohort of patients with vWD-2b ("vWD-2b
Cohort") will begin enrollment at the commencement of the study and continue independently
of the course of the TTP Remission Cohorts. Up to 4 patients will be included in each of the
TTP cohorts. The vWD-2b Cohort is expected to consist of up to 12 vWD-2b patients.
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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