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Study of HRO761 Alone or in Combination in Cancer Patients With Specific DNA Alterations Called Microsatellite Instability or Mismatch Repair Deficiency.

Phase 1
Recruiting
Conditions
MSIhi or dMMR Advanced Unresectable or Metastatic Solid Tumors, Including Colorectal Cancers
Interventions
Registration Number
NCT05838768
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

The main purpose of the study is to evaluate the safety and tolerability of HRO761 and identify the recommended dose(s), i.e., the optimal safe and active dose of HRO761 alone or in combination with tislelizumab or irinotecan that can be given to patients who have cancers with specific molecular alterations called MSIhi (Microsatellite Instability-high) or dMMR (Mismatch Repair Deficient) that might work best to treat these specific cancer types and to understand how well HRO761 is able to treat those cancers.

Detailed Description

The new drug being tested in the study, HRO761, is an oral drug that acts on a protein called Werner (WRN), which may contribute to cancer growth. By acting on WRN, HRO761 may be able to stop the growth of the cancer.

This is the first time HRO761 is given to patients and the first time HRO761 is used in combination with tislelizumab or irinotecan.

Tislelizumab has been used in other cancer studies in the past few years and irinotecan is a drug approved in several countries and is used as standard treatment for certain types of cancer (e.g., colon cancer and small cell lung cancer).

This research study will consist of various treatment arms to investigate HRO761 as single agent and in the combinations.

For HRO761 single agent, the research will be done in two parts the first part is called "dose escalation" and the second part is called "dose optimization" In the dose escalation part, different groups of people will be given different doses of HRO761 to understand how the body reacts to different doses of the drug and how well the drug acts against the cancer. During the dose optimization part, the selected doses will be tested in more patients until a recommended dose(s) is found.

The combinations of HRO761with tislelizumab or irinotecan will also first be tested in a dose escalation part to find the recommended doses of HRO761 in these combinations.

Once the recommended doses are determined, more people may be treated with HRO761 alone or together with tislelizumab or irinotecan to further assess the study treatment effects against various types of MSIhi or dMMR cancers. This part is called dose expansion.

For this research, a number of blood and tissue samples will be collected during the study. Patients may be asked to come approximately 8 times to the clinic during the first 8 weeks and approximately every 2 or 4 weeks thereafter.

Patients will be in the study as long as their study doctor believes that they may be benefiting from the study treatment, unless the patient decides to stop study treatment.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
327
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
A: HRO761 single agentHRO761phase Ib (Dose finding (Escalation and Optimization) and expansion)
B: HRO761 + pembrolizumabHRO761phase Ib (Dose escalation and expansion)
B: HRO761 + pembrolizumabpembrolizumabphase Ib (Dose escalation and expansion)
C: HRO761 + irinotecanHRO761phase Ib (Dose escalation and expansion)
C: HRO761 + irinotecanirinotecanphase Ib (Dose escalation and expansion)
Primary Outcome Measures
NameTimeMethod
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)at month 36

Month 36 is assumed to be study end.

Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs) and laboratory results qualifying and reported as AEs.

Frequency of dose reductions as a measure of tolerabilityat month 36

Month 36 is assumed to be study end

Number of dose reductions by treatment group/arm as a measure of tolerability.

Incidence of dose limiting toxicities (DLTs) of treatment (Escalation only)at Day 28

A DLT is defined as an adverse event or abnormal laboratory value that is not primarily related to disease, disease progression, intercurrent illness/injury, or concomitant medications that occurs within the first 28 days of study treatment and meets a defined criteria.

Frequency of dose interuptions as a measure of tolerabilityat month 36

Month 36 is assumed to be study end

Number of dose interruptions by treatment group/arm as a measure of tolerability.

Frequency of dose discontinuations as a measure of tolerabilityat month 36

Month 36 is assumed to be study end

Number of dose discontinuations by treatment group/arm as a measure of tolerability.

Secondary Outcome Measures
NameTimeMethod
Overall Response Rate (ORR) per RECIST v1.1at month 36

Month 36 is assumed to be study end

ORR is the percentage of patients with a best overall response of Complete Response (CR) or Partial Response (PR).

Disease Control Rate (DCR) per RECIST v1.1at month 36

Month 36 is assumed to be study end

DCR is the percentage of patients with a best overall response of CR or PR or Stable Disease (SD)

Progression Free Survival (PFS) per RECIST v1.1at month 36

Month 36 is assumed to be study end

PFS is defined as the time from the date of start of treatment to the date of the first documented progression or death due to any cause.

Duration of Response (DOR) per RECIST v1.1at month 36

Month 36 is assumed to be study end

DOR is the time between the date of first documented response (CR or PR) and the date of progression or death due to any cause.

Plasma concentrations of HRO761at Day 1, Day 8, Day 29, Day 57, Day 85, Day 113, Day 141, Day 225, Day 309 and EOT

Plasma concentrations of HRO761 will be measured using a validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) assay.

PK parameter (Tmax) of HRO761at month 12

Cycle 12 (the duration of 1 cycle is 28 days).

Time to maximum observed concentration (Tmax) determined by non-compartmental PK analysis of plasma concentration-time profiles HRO761.

PK parameter (Cmax) of HRO761at month 12

Cycle 12 (the duration of 1 cycle is 28 days).

Maximum observed concentration (Cmax) determined by non-compartmental pharmacokinetic (PK) analysis of plasma concentration-time profiles HRO761.

PK parameter (AUC) of HRO761at month 12

Cycle 12 (the duration of 1 cycle is 28 days).

Area under the plasma concentration-time curve (AUC) determined by non-compartmental PK analysis of plasma concentration-time profiles HRO761.

Trial Locations

Locations (7)

Novartis Investigative Site

🇬🇧

Oxford, United Kingdom

University of California LA

🇺🇸

Los Angeles, California, United States

UCSF

🇺🇸

San Francisco, California, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Memorial Sloan Kettering

🇺🇸

New York, New York, United States

Columbia University Medical Ctr

🇺🇸

New York, New York, United States

Univ of TX MD Anderson Cancer Cntr

🇺🇸

Houston, Texas, United States

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