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First-in-Human Study of OKI-219 in Advanced Solid Tumors and Advanced Breast Cancer

Phase 1
Recruiting
Conditions
Advanced Cancer
Breast Cancer
Advanced Solid Tumors
PI3K Gene Mutation
Interventions
Registration Number
NCT06239467
Lead Sponsor
OnKure, Inc.
Brief Summary

OKI-219-101 is a Phase 1a/1b, open-label, multicenter, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of OKI-219 as monotherapy and in combination with other anti-cancer drugs. Phase 1a (Part A) will investigate escalating doses of OKI-219 monotherapy, and Phase 1b will investigate OKI-219 (at a tolerated dose determined in Part A) in combination with fulvestrant (Part B), trastuzumab and tucatinib (Part C), atirmociclib (Part D), and ribociclib and fulvestrant (Part E). Participants will continue to receive study treatment until disease progression, intolerable toxicity, or other study treatment withdrawal criteria are met.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
200
Inclusion Criteria
  • Participants with advanced solid tumors with documented evidence of a PI3KαH1047R mutation in tumor tissue and/or blood (ie, ctDNA).
  • Eastern Cooperative Oncology Group (ECOG) Performance status score of to 1.
  • Life expectancy > 12 weeks for Part A and > 6 months for Parts B, C, D, and E in the opinion of the Investigator.
  • Adequate organ and bone marrow function
  • Have adequate archival tumor tissue sample available or be approved by the Sponsor for enrollment if no tumor sample is available.
  • At least 1 measurable lesion based on RECIST version 1.1.

Additional Cohort-specific key inclusion criteria:

Part A

  • Participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer, must have received at least 1 prior line of hormonal therapy and at least 1 prior line of CDK4/6-inhibitor in the advanced or metastatic setting.
  • Participants with HER2+ locally advanced, unresectable or metastatic breast cancer, must have received prior taxane, trastuzumab, pertuzumab, and tucatinib. Prior trastuzumab deruxtecan is allowed but not required.
  • Participants with HER2-low breast cancer must have received prior trastuzumab deruxtecan.
  • Participants with colorectal cancer must have KRAS wild-type disease.

Part B

  • Participants with locally advanced, unresectable or metastatic HR+/HER2- breast cancer must have received at least 1 prior line of hormonal therapy in the advanced or metastatic setting and at least 1 prior CDK4/6-inhibitor.
  • Participants with HER2-low breast cancer should have received prior trastuzumab deruxtecan

Part C ● Participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer must have received prior taxane, trastuzumab, and pertuzumab unless unavailable in the region or contraindicated. Prior trastuzumab deruxtecan is allowed but not required.

Part D

● Participants must have HR+/HER2- locally advanced, unresectable or metastatic breast cancer

Part E ● Participants must have HR+/HER2- locally advanced, unresectable or metastatic breast cancer.

Key

Exclusion Criteria
  • Treatment with any investigational product or other anticancer therapy within 28 days or 5 half-lives, whichever is shorter, of the start of treatment
  • Participants with a known KRAS mutation.
  • Participants with a known deleterious mutation in phosphatase and tensin homolog (PTEN) or negative for PTEN protein expression by IHC.
  • Major surgery or wide-field radiation within 28 days or limited field palliative radiation within 7 days prior to the first dose of study drug.
  • Known active central nervous system metastasis, including leptomeningeal disease.
  • Uncontrolled Type 1 or Type 2 diabetes as defined by HbA1C ≥ 8%.
  • Concomitant active malignancy or previous malignancy within 2 years of the time of enrollment.
  • Impaired cardiovascular function or clinically significant cardiovascular disease,
  • History of symptomatic drug-induced pneumonitis.
  • Participants with active HIV, Hepatitis B, and Hepatitis C viral infections

Additional Cohort-specific key exclusion criteria:

Part C:

  • Grade 2 or higher diarrhea at study entry.
  • History of chronic liver disease.

Part E:

● History of interstitial lung disease.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Phase 1a: Part A Dose EscalationOKI-219OKI-219 Monotherapy Dose Escalation in participants with advanced solid tumors with the PI3KαH1047R mutation
Phase 1b: Part B Dose EscalationOKI-219OKI-219 + Fulvestrant Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part B Dose EscalationFulvestrantOKI-219 + Fulvestrant Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part B Dose OptimizationOKI-219OKI-219 + Fulvestrant Dose Optimization in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part B Dose OptimizationFulvestrantOKI-219 + Fulvestrant Dose Optimization in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose EscalationOKI-219OKI-219 + Tucatinib + Trastuzumab Dose Escalation in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose EscalationTrastuzumabOKI-219 + Tucatinib + Trastuzumab Dose Escalation in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose EscalationTucatinibOKI-219 + Tucatinib + Trastuzumab Dose Escalation in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose ExpansionOKI-219OKI-219 + Tucatinib + Trastuzumab Dose Expansion in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose ExpansionTrastuzumabOKI-219 + Tucatinib + Trastuzumab Dose Expansion in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part C Dose ExpansionTucatinibOKI-219 + Tucatinib + Trastuzumab Dose Expansion in participants with HR±/HER2+ locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose EscalationOKI-219OKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose EscalationFulvestrantOKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose EscalationAtirmociclibOKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose ExpansionOKI-219OKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose ExpansionFulvestrantOKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part D Dose ExpansionAtirmociclibOKI-219 + Fulvestrant + Atirmociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose EscalationOKI-219OKI-219 + Fulvestrant + Ribociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose EscalationFulvestrantOKI-219 + Fulvestrant + Ribociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose EscalationRibociclibOKI-219 + Fulvestrant + Ribociclib Dose Escalation in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose ExpansionOKI-219OKI-219 + Fulvestrant + Ribociclib Dose Expansion in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose ExpansionFulvestrantOKI-219 + Fulvestrant + Ribociclib Dose Expansion in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Phase 1b: Part E Dose ExpansionRibociclibOKI-219 + Fulvestrant + Ribociclib Dose Expansion in participants with HR+/HER2- locally advanced, unresectable or metastatic breast cancer with the PI3KαH1047R mutation
Primary Outcome Measures
NameTimeMethod
Identify maximum tolerated dose (MTD) of OKI-219 in monotherapyCycle 1 (First 28 days on treatment)

Frequency of participants experiencing dose-limiting toxicities during the first 28-day cycle

Assess safety of OKI-219 as monotherapy or in combination with other anti-cancer therapies: incidence of SAEsThrough 30 days after last dose, an average of 1 year

Number and type of SAEs experienced by participants during treatment and follow-up

Assess safety of OKI-219 as monotherapy or in combination with other anti-cancer therapies: incidence of Grade 2 or greater treatment emergent adverse eventsThrough 30 days after last dose, an average of 1 year

Number of treatment-emergent adverse events (TEAEs) equal or greater than Grade 2 experienced during treatment and follow-up

Assess rate of dose modifications during treatment with OKI-219 as monotherapy or in combination with other anti-cancer therapiesThrough last study dose, an average of 1 year

rate of dose modifications

Secondary Outcome Measures
NameTimeMethod
Assess the plasma PK of OKI-219 following single and multiple doses as monotherapy or in combination with other anti-cancer therapies: maximum plasma concentration (Cmax)Through cycle 6 of treatment (up to 28 weeks)

PK of OKI-219: Cmax

Assess the plasma PK of OKI-219 following single and multiple doses as monotherapy or in combination with other anti-cancer therapies: time of maximum plasma concentration (Tmax)Through cycle 6 of treatment (up to 28 weeks)

PK of OKI-219: Tmax

Assess the plasma PK of OKI-219 following single and multiple doses as monotherapy or in combination with other anti-cancer therapies: area under the plasma concentration-time curve (AUC)Through cycle 6 of treatment (up to 28 weeks)

PK of OKI-219: AUC

Assess the plasma PK of OKI-219 following single and multiple doses as monotherapy or in combination with other anti-cancer therapies: terminal elimination half-life time (t1/2)Through cycle 6 of treatment (up to 28 weeks)

PK of OKI-219: t1/2

To estimate the preliminary antitumor activity of OKI-219 as monotherapy and in combination with other anti-cancer therapies: objective response rate (ORR)Up to approximately 36 months

ORR per investigator assessed Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1)

To estimate the preliminary antitumor activity of OKI-219 as monotherapy and in combination with other anti-cancer therapies: clinical benefit rate (CBR)Up to approximately 36 months

CBR per investigator assessed Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1)

Dose optimization only: to estimate the preliminary antitumor activity of OKI-219 as monotherapy and in combination with other anti-cancer therapies: progression free survival (PFS)Up to approximately 36 months

PFS per investigator assessed Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1)

To assess the dose-response impact of OKI-219 as monotherapy and in combination with other anti-cancer therapies on PI3KαH1047R ctDNA levelsThrough last study dose, an average of 1 year

Changes in PI3KαH1047R ctDNA on treatment and end of treatment (EOT) compared to baseline.

To determine the impact of OKI-219 dosing as monotherapy and in combination with other anti-cancer therapies on blood glucose and insulinThrough last study dose, an average of 1 year

Changes in plasma glucose, serum insulin, serum c-peptide levels, and hemoglobin A1c (HbA1c) will be evaluated on treatment compared to baseline.

To assess the PDx activity of OKI-219 as monotherapy and in combination with other anti-cancer therapiesThrough last study dose, an average of 1 year

PDx activity will be evaluated with serial tumor biopsy samples assessed for PI3K/AKT/mTOR downstream pathway changes.

Trial Locations

Locations (34)

California Cancer Associates for Research and Excellence

🇺🇸

Encinitas, California, United States

University of California San Diego UCSD

🇺🇸

La Jolla, California, United States

UCLA Jonsson Comprehensive Cancer Center

🇺🇸

Los Angeles, California, United States

Hoag - Huntington Beach

🇺🇸

Newport Beach, California, United States

Regents of the University of Colorado

🇺🇸

Aurora, Colorado, United States

Sarah Cannon Research Institute at HealthONE

🇺🇸

Denver, Colorado, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Karmanos Cancer Insitute

🇺🇸

Detroit, Michigan, United States

Comprehensive Cancer Centers of Nevada

🇺🇸

Las Vegas, Nevada, United States

Stony Brook University

🇺🇸

Stony Brook, New York, United States

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California Cancer Associates for Research and Excellence
🇺🇸Encinitas, California, United States
Dante Pasiliao
Contact
dpasiliao@ccare.com

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