A Phase 2 Study of ONC206 in Advanced Pheochromocytoma and Paraganglioma
Summary
This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).
Detailed description
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.
Arms & interventions
- DrugONC206
150 mg BID TIW
- DrugONC206: Dose 1
- DrugONC206: Dose 2
Outcome measures
Primary
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.
Secondary
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
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.
Eligibility criteria
Study locations (9)
UCLA
Los Angeles, California, 90095
Stanford
Palo Alto, California, 94604
U of Colorado
Aurora, Colorado, 80045
U of Michigan
Ann Arbor, Michigan, 48109
Mayo-Rochester
Rochester, Minnesota, 55905
Washington University
St Louis, Missouri, 63110
Columbia University Medical Center
New York, New York, 10032
PENN
Philadelphia, Pennsylvania, 19104
UTAH
Salt Lake City, Utah, 84112