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NCT Number: NCT06898450

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

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.

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Key information

Conditions

Advanced Solid Tumors Cancer Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Genetic Diseases, Inborn Genomic Instability MSI-H Cancer Metabolic Diseases Microsatellite Instability Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Werner Syndrome

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Liverpool Hospital, Liverpool, New South Wales, Australia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

NDI-219216

Drug

NDI-219216 is a highly selective small molecule inhibitor of WRN helicase activity.

Primary outcomes

  1. Part A Primary Objective: Incidence of dose limiting toxicities (DLTs)

    Time frame: 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)

  2. Part A Primary Outcome: • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs), according to NCI CTCAE v5.0

    Time frame: From 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.

  3. Part A Primary Outcome: Incidence and severity of Treatment Emergent Adverse Events (TEAEs) and Treatment Related Adverse Events (TRAEs) as assessed by the Investigator

    Time frame: From 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).

  4. Part B Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.

    Time frame: From start of study treatment until end of follow-up, up to approximately 18 months. Each Cycle is 28 days.

  5. Part B Primary Outcome: Duration of Response (DOR) per RECIST v1.1

    Time frame: 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 18 months. Each Cycle is 28 days.

  6. Part B Primary Outcome: Incidence and severity of AEs according to NCI CTCAE v5.0.

    Time frame: 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).

  7. Part C Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.

    Time frame: From start of study treatment until end of follow-up, up to approximately 17 months. Each Cycle is 28 days.

  8. Part C Primary Outcome: Duration of Response (DOR) per RECIST v1.1.

    Time frame: 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 outcomes

  1. Part A Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.

    Time frame: 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.

  2. Part A Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216

    Time frame: 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.

  3. Part A Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216

    Time frame: 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.

  4. Part A Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216

    Time frame: At 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.

  5. Part B Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.

    Time frame: 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.

  6. Part B Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216

    Time frame: 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.

  7. Part B Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216

    Time frame: 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.

  8. Part B Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216

    Time frame: At 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.

  9. Part C Secondary Objective: Incidence and severity of AEs according to NCI CTCAE v5.0.

    Time frame: 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).

  10. Part C Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.

    Time frame: 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.

  11. Part C Secondary Objective: Maximum Plasma Concentration Observed (Cmax) of NDI-219216

    Time frame: 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.

  12. Part C Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216

    Time frame: 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.

  13. Part C Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Katie Ard, MSN

CONTACT

[email protected]

303-646-7297

Sean Rossi

CONTACT

[email protected]

857-600-8779

Sponsors and collaborators

Lead sponsor

Nimbus Wadjet, Inc.

Industry

Collaborators

  • Worldwide Clinical Trials

Registry information

Official study title

A Phase 1 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Antitumor Activity of NDI-219216 in Patients With Advanced Solid Tumors With/Without Microsatellite Instability and/or Deficient Mismatch Repair

Important dates

Study start
2025
Primary completion
2031
Study completion
2031
First posted
Mar 27, 2025
Registry last updated
May 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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