This is a two-part Phase 1, open label, multi-center, single arm, non-randomized, multiple dose, safety, pharmacokinetic (PK) and preliminary efficacy study of single agent NST-628 in adult patients with MAPK pathway mutated/dependent advanced solid tumors who have exhausted standard treatment options.
The study includes two parts, a dose escalation part (Part A) followed by a dose expansion part (Part B). Part A will estimate the maximum tolerated dose (MTD) in dose escalation cohorts in patients with advanced solid tumors for whom no standard therapy is available in order to establish the recommended dose for expansion (RDE). Successive cohorts of subjects will receive escalating doses of NST-628 orally once daily in 28-day cycles. Bayesian Optimal Interval (BOIN) method will be used for dose escalation. Once MTD is reached or dose escalation is stopped prior to reaching MTD and provisional RDE selected, the provisional RDE level will be expanded. If warranted by dose/toxicity/anti-tumor activity observations, additional, lower dose level(s) may also be expanded. Part B of the study will include up to 6 cohorts of approximately up to 30 subjects each with select MAPK pathway mutant solid tumors enrolled at the RDE in order to explore benefit from treatment as suggested by preclinical findings and will better define the safety profile of NST-628 at the RDE. Additional safety information gathered in Part B may be used to modify the dose recommended for future studies. The end of the study is the last visit of the last subject.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
230
NST-628 is a small molecule non-covalent pan-RAF/MEK dual molecular glue targeting RAF and MEK nodes of MAPK pathway.
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States
RECRUITINGUCLA Hematology/Oncology
Westwood, Los Angeles, California, United States
RECRUITINGSarah Cannon Research Institute at Health ONE
Denver, Colorado, United States
RECRUITINGYale Cancer Center
New Haven, Connecticut, United States
RECRUITINGMoffitt Cancer Center
Tampa, Florida, United States
RECRUITINGRoswell Park
Buffalo, New York, United States
RECRUITINGLaura & Isaac Perlmutter Cancer Center at NYU Langone Health
New York, New York, United States
RECRUITINGColumbia University Medical Center
New York, New York, United States
RECRUITINGMemorial Slone Kettering Cancer Center
New York, New York, United States
RECRUITINGUPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, United States
RECRUITING...and 13 more locations
Part A and B: Evaluate the safety of NST-628 in patients with advanced solid tumors
Adverse effects
Time frame: Through study completion, an average of 1 year
Part A: Determine the recommended dose for expansion of NST-628
Dose limiting toxicities (DLTs)
Time frame: The first 28 days of treatment (DLTs)
Part B: Evaluate objective tumor response rate
Objective response per RECIST v. 1.1 or other response assessment tool standard for a given tumor type.
Time frame: Through study completion, an average of 1 year
Part A: Evaluate objective tumor response rate
Objective response per RECIST v. 1.1 or other response assessment tool standard for a given tumor type.
Time frame: Through study completion, an average of 1 year
Part A and B: Evaluate progression free survival (PFS)
PFS defined as the time to first occurrence of disease progression per RECIST v1.1 (or other response assessment tool standard for a given tumor type) or death
Time frame: Through study completion, an average of 1 year
Part A and B: Evaluate overall survival (OS)
Overall survival (OS) defined as the time to death
Time frame: Through study completion, an average of 2 years
Part A and B: Characterize the pharmacokinetics of NST-628
NST-628 concentrations in plasma
Time frame: Through study completion, an average of 1 year
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