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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06898450
Registration number
NCT06898450
Ethics application status
Date submitted
10/03/2025
Date registered
27/03/2025
Date last updated
3/08/2025
Titles & IDs
Public title
A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.
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Scientific title
A Phase 1/2 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
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Secondary ID [1]
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9216-101
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Advanced Solid Tumors Cancer
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MSI-H Cancer
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - NDI-219216
Experimental: Part A dose escalation - Part 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.
Experimental: Part B Project Optimus - Part 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.
Experimental: Part C Dose Expansion - Part 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.
Treatment: Drugs: NDI-219216
NDI-219216 is a highly selective small molecule inhibitor of WRN helicase activity.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Part A Primary Objective: Incidence of dose limiting toxicities (DLTs)
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Assessment method [1]
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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)
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Timepoint [1]
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The first 21 days of Cycle 1 (Cycle 1 is 28 days).
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Primary outcome [2]
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Part A Primary Outcome: • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs), according to NCI CTCAE v5.0
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Assessment method [2]
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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.
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Timepoint [2]
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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.
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Primary outcome [3]
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Part A Primary Outcome: Incidence and severity of Treatment Emergent Adverse Events (TEAEs) and Treatment Related Adverse Events (TRAEs) as assessed by the Investigator
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Assessment method [3]
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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).
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Timepoint [3]
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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.
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Primary outcome [4]
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Part B Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.
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Assessment method [4]
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Timepoint [4]
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From start of study treatment until end of follow-up, up to approximately 18 months. Each Cycle is 28 days.
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Primary outcome [5]
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Part B Primary Outcome: Duration of Response (DOR) per RECIST v1.1
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Assessment method [5]
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Timepoint [5]
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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.
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Primary outcome [6]
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Part B Primary Outcome: Incidence and severity of AEs according to NCI CTCAE v5.0.
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Assessment method [6]
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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).
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Timepoint [6]
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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.
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Primary outcome [7]
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Part C Primary Objective: Overall Response Rate (ORR) per RECIST v1.1.
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Assessment method [7]
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Timepoint [7]
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From start of study treatment until end of follow-up, up to approximately 17 months. Each Cycle is 28 days.
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Primary outcome [8]
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Part C Primary Outcome: Duration of Response (DOR) per RECIST v1.1.
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Assessment method [8]
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Timepoint [8]
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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.
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Secondary outcome [1]
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Part A Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.
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Assessment method [1]
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Timepoint [1]
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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.
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Secondary outcome [2]
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Part A Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216
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Assessment method [2]
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Timepoint [2]
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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.
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Secondary outcome [3]
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Part A Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216
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Assessment method [3]
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Timepoint [3]
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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.
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Secondary outcome [4]
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Part A Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216
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Assessment method [4]
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Timepoint [4]
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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.
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Secondary outcome [5]
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Part B Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.
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Assessment method [5]
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Timepoint [5]
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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.
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Secondary outcome [6]
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Part B Secondary Outcome: Maximum Plasma Concentration Observed (Cmax) of NDI-219216
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Assessment method [6]
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Timepoint [6]
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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.
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Secondary outcome [7]
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Part B Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216
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Assessment method [7]
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Timepoint [7]
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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.
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Secondary outcome [8]
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Part B Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216
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Assessment method [8]
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Timepoint [8]
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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.
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Secondary outcome [9]
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Part C Secondary Objective: Incidence and severity of AEs according to NCI CTCAE v5.0.
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Assessment method [9]
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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).
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Timepoint [9]
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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.
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Secondary outcome [10]
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Part C Secondary Objective: Plasma concentrations of NDI-219216 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS) assay.
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Assessment method [10]
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Timepoint [10]
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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.
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Secondary outcome [11]
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Part C Secondary Objective: Maximum Plasma Concentration Observed (Cmax) of NDI-219216
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Assessment method [11]
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Timepoint [11]
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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.
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Secondary outcome [12]
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Part C Secondary Outcome: Time of Maximum Plasma Concentration Observed (Tmax) of NDI-219216
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Assessment method [12]
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Timepoint [12]
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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.
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Secondary outcome [13]
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Part C Secondary Outcome: Area Under the Plasma Concentration-Time Curve (AUC0-last) of NDI-219216
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Assessment method [13]
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Timepoint [13]
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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.
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Eligibility
Key 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).
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Minimum age
18
Years
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Maximum age
99
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Clinically significant cardiovascular disease.
* Patients with known WRN syndrome.
* Pregnancy, breastfeeding, or intention of becoming pregnant during the study.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
31/03/2025
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2031
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Actual
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Sample size
Target
134
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,SA
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Recruitment hospital [1]
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Liverpool Hospital - Liverpool
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Recruitment hospital [2]
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Southern Oncology Clinical Research Unit - Bedford Park
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Recruitment postcode(s) [1]
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2170 - Liverpool
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Recruitment postcode(s) [2]
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5042 - Bedford Park
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Illinois
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Country [3]
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United States of America
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State/province [3]
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Kentucky
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Country [4]
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United States of America
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State/province [4]
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New York
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Country [5]
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United States of America
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State/province [5]
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Ohio
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Country [6]
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United States of America
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State/province [6]
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Rhode Island
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Country [7]
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United States of America
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State/province [7]
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South Carolina
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Country [8]
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United States of America
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State/province [8]
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Virginia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Nimbus Wadjet, Inc.
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Address
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Other collaborator category [1]
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Other
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Name [1]
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Worldwide Clinical Trials
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Address [1]
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT06898450
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Anita Scheuber, MD, PhD
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Address
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Nimbus Therapeutics, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Sean Rossi
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Address
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Country
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Phone
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857-600-8779
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
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No/undecided IPD sharing reason/comment
It is not yet known if there will be a plan to make IPD available.
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06898450
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