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A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.

Phase 1
Recruiting
Conditions
Advanced Solid Tumors Cancer
MSI-H Cancer
Interventions
Drug: NDI-219216
Registration Number
NCT06898450
Lead Sponsor
Nimbus Wadjet, Inc.
Brief Summary

The goal of this clinical trial is to learn if NDI-219216 is safe for patients, and if NDI-219216 might be a possible treatment for advanced solid tumors in the later phases of the study.

The main questions it aims to answer are:

Is NDI-219216 safe and what kinds of side effects might it cause? What kind of effects does NDI-219216 have on the body? Does NDI-219216 have any impact on tumor size?

Participants will:

Take NDI-219216 every day by mouth. Visit the clinic 6 times during Cycle 1, 2 times during Cycle 2, once a month thereafter for checkups and tests while on the study, then one time for an end of treatment visit. After the End of Study, a follow up will occur but can be done on the phone.

Keep a diary of their tablet consumption and symptoms experienced.

Detailed Description

Study 9216-101 is a first-in-human (FIH), Phase 1/2, open-label, dose escalation, dose optimization, and dose expansion study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of NDI-219216 in patients with advanced solid tumors.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
134
Inclusion Criteria
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1
  • Have unresectable and/or metastatic solid tumors (with or without MSI-H/dMMR) refractory to or intolerant to previous SoC therapy or for which no SoC therapy exists
  • Presence of measurable disease according to RECIST version 1.1 except for Part A (Dose Escalation)
  • Adequate bone marrow / hematologic, end-organ, and cardiovascular function
  • Resolution of all acute (or toxic) adverse effects of prior therapies, radiation therapy, or surgical procedures to Grade ≤ 1 (except fatigue, alopecia, and peripheral neuropathy).
Exclusion Criteria
  • Clinically significant cardiovascular disease.
  • Patients with known WRN syndrome.
  • Pregnancy, breastfeeding, or intention of becoming pregnant during the study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part A dose escalationNDI-219216Part A Dose Escalation will involve enrolling sequential cohorts with increasing doses of NDI-219216 administered daily in repeating 28-day treatment cycles. The Dose Limiting Toxicity review period for each cohort will be 21 days for each patient enrolled, with review by a Safety Review Committee prior to escalation to the next dose level.
Part B Project OptimusNDI-219216Part B will enroll up to 3 cohorts of patients randomized between up to 3 dose levels determined from Part A Dose Escalation. NDI-219216 will be administered daily in repeating 28-day treatment cycles.
Part C Dose ExpansionNDI-219216Part C will enroll 2 groups of patients with dMMR/MSI-h status and other select criteria, utilizing the optimal dose identified from Part B. NDI-219216 will be administered daily in 28-day repeating cycles.
Primary Outcome Measures
NameTimeMethod
Part A Primary Objective: Incidence of dose limiting toxicities (DLTs)The first 21 days of Cycle 1 (Cycle 1 is 28 days).

Assessments will include electrocardiograms (ECGs), echocardiogram, cardiac biomarker troponin I, physical examination, vital signs (including blood pressure, pulse), and evaluation of laboratory parameters (clinical chemistry, hematology, and coagulation)

Part A Primary Outcome: • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs), according to NCI CTCAE v5.0From first dose of study drug until 30 days after last dose of study drug; up to approximately 11-12 months. Each Cycle is 28 days.

Assessments will include standard electrocardiograms (ECGs), echocardiogram, cardiac biomarker troponin I, physical examination, vital signs (including blood pressure, pulse), and evaluation of laboratory parameters (clinical chemistry, hematology, and coagulation.

Part A Primary Outcome: Incidence and severity of Treatment Emergent Adverse Events (TEAEs) and Treatment Related Adverse Events (TRAEs) as assessed by the InvestigatorFrom first dose of study drug until 30 days after last dose of study drug; up to approximately 11-12 months. Each Cycle is 28 days.

Assessments will include standard electrocardiograms (ECGs), echocardiogram, cardiac biomarker troponin I, physical examination, vital signs (including blood pressure, pulse), and evaluation of laboratory parameters (clinical chemistry, hematology, and coagulation).

Part B Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.From start of study treatment until end of follow-up, up to approximately 18 months. Each Cycle is 28 days.
Part B Primary Outcome: Duration of Response (DOR) per RECIST v1.1From the time of first occurrence of a documented response until the time of documented disease progression or death from any cause, whichever occurs first; up to approximately 18 months. Each Cycle is 28 days.
Part B Primary Outcome: Incidence and severity of AEs according to NCI CTCAE v5.0.From first dose of study drug until 30 days after last dose of study drug; up to approximately 18 months. Each Cycle is 28 days.

Assessments will include standard electrocardiograms (ECGs), echocardiogram, cardiac biomarker troponin I, physical examination, vital signs (including blood pressure, pulse), and evaluation of laboratory parameters (clinical chemistry, hematology, and coagulation).

Part C Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.From start of study treatment until end of follow-up, up to approximately 17 months. Each Cycle is 28 days.
Part C Primary Outcome: Duration of Response (DOR) per RECIST v1.1.From the time of first occurrence of a documented response until the time of documented disease progression or death from any cause, whichever occurs first, up to approximately 17 months. Each Cycle is 28 days.
Secondary Outcome Measures
NameTimeMethod
Part A Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part A Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part A Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part A Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1 from time zero to the last observable concentration. Cycle 1 is 28 days in length.
Part B Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part B Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part B Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part B Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1 from time zero to the last observable concentration. Cycle 1 is 28 days in length.
Part C Secondary Objective: Incidence and severity of AEs according to NCI CTCAE v5.0.From first dose of study drug until 30 days after last dose of study drug; up to approximately 17 months. Each Cycle is 28 days.

Assessments will include standard electrocardiograms (ECGs), echocardiogram, cardiac biomarker troponin I, physical examination, vital signs (including blood pressure, pulse), and evaluation of laboratory parameters (clinical chemistry, hematology, and coagulation).

Part C Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part C Secondary Objective: Maximum Plasma Concentration Observed (Cmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part C Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.
Part C Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216At pre-specified timepoints on Day 1, Day 2, Day 8, Day 21, and Day 22 of Cycle 1. Cycle 1 is 28 days in length.

Trial Locations

Locations (11)

USC San Diego Health

🇺🇸

La Jolla, California, United States

USC Norris Comprehensive Cancer Center

🇺🇸

Los Angeles, California, United States

University of Chicago Medicine

🇺🇸

Chicago, Illinois, United States

University of Louisville James Graham Brown Cancer Center

🇺🇸

Louisville, Kentucky, United States

Cayuga Cancer Center

🇺🇸

Ithaca, New York, United States

Taylor Cancer Research Center

🇺🇸

Maumee, Ohio, United States

Brown University Health

🇺🇸

Providence, Rhode Island, United States

Prisma Health Cancer Institute - Multidisciplinary Center

🇺🇸

Greenville, South Carolina, United States

Tennessee Oncology

🇺🇸

Nashville, Tennessee, United States

University of Virginia Emily Couric Clinical Cancer Center

🇺🇸

Charlottesville, Virginia, United States

Virginia Cancer Specialists, P.C. - Fairfax

🇺🇸

Fairfax, Virginia, United States

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