This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).
Two-Stage Study to evaluate ONC206 as monotherapy doses: Stage 1: Participants will receive 150mg ONC206 twice daily, on three consecutive days per week (BID TIW) in each 28-day cycle. * Part A: Initial number of participants will be monitored. * Part B: If a set number of participants have a response, then more participants will be enrolled and treated. Stage 2: If Stage 1 meets the planned response, the study will proceed, and participants will be randomized 1:1 to receive 1 of 2 ONC206 dose levels.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
90
UCLA
Los Angeles, California, United States
Stanford
Palo Alto, California, United States
U of Colorado
Aurora, Colorado, United States
Miami Cancer Institute
Miami, Florida, United States
Overall Response Rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
ORR defined as the number of participants with a confirmed complete response (CR) or partial response during the study, as per RECIST v1.1.
Time frame: Up to 36 months.
Duration of Response (DOR)
Time frame: Up to 36 months.
Time to Response (TTR)
Time frame: Up to 36 months.
Disease Control Rate (DCR)
Time frame: Up to 36 months.
Progression Free Survival (PFS) by RECIST v1.1
Time frame: Up to 36 months.
Overall Survival (OS)
Time frame: Up to 36 months.
Change from Baseline in Antihypertensive Medication Dose
Time frame: Up to 36 months.
Change from Baseline in Biochemical Response (Metanephrines/Disease Markers)
Time frame: Up to 36 months.
Incidence of Adverse Events (AEs)
Time frame: Up to 36 months.
Number of Participants with Clinically Significant Changes from Baseline in Clinical Laboratory Parameters
Time frame: Up to 36 months.
Number of Participants with Grade 4+ Clinical Laboratory Parameters
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U of Michigan
Ann Arbor, Michigan, United States
Mayo-Rochester
Rochester, Minnesota, United States
Washington University
St Louis, Missouri, United States
Columbia University Medical Center
New York, New York, United States
PENN
Philadelphia, Pennsylvania, United States
UTAH
Salt Lake City, Utah, United States
Time frame: Up to 36 months.
Number of Participants with Clinically Significant Changes from Baseline in Electrocardiogram (ECG) Parameters
Time frame: Up to 36 months.
Maximum Observed Concentration of ONC206
Time frame: Up to 36 months.
Time of Maximum Observed Concentration (Tmax) of ONC206
Time frame: Up to 36 months.
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUCinf)
Time frame: Up to 36 months.
Area Under the Concentration Time Curve Over the Dosing Interval
Time frame: Up to 36 months.
Terminal Half-Life (t1/2) of ONC206
Time frame: Up to 36 months.
Change from Baseline in European Organization for the Research and Treatment of Cancer Quality of Life-Core Questionnaire (EORTC-QLQ-C30)
Time frame: Up to 36 months.
Change from Baseline in Functional Assessment of Cancer Therapy - Bone Pain (FACT-BP)
Time frame: Up to 36 months.