KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera

Status:	Recruiting
Conditions:	Cancer, Hematology
Therapuetic Areas:	Hematology, Oncology
Healthy:	No
Age Range:	18 - Any
Updated:	4/3/2019
Start Date:	January 15, 2019
End Date:	October 2023
Contact:	John Mei
Email:	jmei@kartosthera.com
Phone:	6505420136

A Two-Part, Randomized, Open-label, Multicenter, Phase 2a/2b Study of the Efficacy, Safety, and Pharmacokinetics of KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera

This study evaluates KRT-232, a novel oral small molecule inhibitor of MDM2, for the
treatment of patients with phlebotomy-dependent polycythemia vera (PV). Inhibition of MDM2 in
PV is a new mechanism of action in PV. In Part A, patients must be resistant or intolerant to
hydroxyurea or have undergone treatment with interferon. In Part B, patients must be
resistant or intolerant to hydroxyurea.

This study is a global, open-label Phase 2a/2b study to determine the efficacy and safety of
KRT-232. In Part A of the study, patients will be randomly assigned to 3 arms with 2
different doses and 2 different dosing schedules of KRT 232. In Part B of the study, patients
will be randomized either to treatment with KRT-232 administered at the recommended dose and
schedule from Part A or to treatment with ruxolitinib.

Inclusion Criteria:

- Confirmed diagnosis of PV (WHO 2016)

- ECOG ≤ 2

- Part A: patients with and without splenomegaly are eligible

- Part A: patients must be resistant or intolerant to hydroxyurea or have undergone
treatment with interferon

- Part B: only patients with splenomegaly are eligible

- Part B: patients must be resistant or intolerant to hydroxyurea

Exclusion Criteria:

- Diagnosis of post-PV myelofibrosis (IWG-MRT)

- Prior treatment with MDM2 inhibitors, p53-directed therapies, HDAC, BCL 2 inhibitors

- Splenic irradiation within 3 months prior to the first dose of study treatment

- Clinically significant thrombosis within 3 months of screening

- Grade 2 or higher QTc prolongation

- Part B: prior treatment with a JAK inhibitor

We found this trial at

sites

Summit Medical Group

Florham Park, New Jersey 07932

from

Florham Park, NJ