A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
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ClinicalTrials.gov Identifier: NCT04659096 |
Recruitment Status :
Completed
First Posted : December 9, 2020
Last Update Posted : December 21, 2022
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Condition or disease | Intervention/treatment | Phase |
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Advanced Solid Tumors | Drug: ION537 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | N/A |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase 1 Trial of ION537 in Patients With Molecularly Selected Advanced Solid Tumors |
Actual Study Start Date : | January 5, 2021 |
Actual Primary Completion Date : | October 19, 2022 |
Actual Study Completion Date : | October 19, 2022 |
Arm | Intervention/treatment |
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Experimental: ION537
Multiple ascending doses of ION537 will be administered by intravenous (IV) injection on Days 1, 4, 8, 11, 15, and 22 in Cycle 1 and weekly dosing in each subsequent cycle until disease progression.
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Drug: ION537
ION537 will be administered by IV injection. |
- Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) and Treatment-emergent Serious Adverse Event (TESAE), Graded by Severity [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Number of Participants With Dose-limiting Toxicities (DLTs) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With Complete Response (CR) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With Partial Response (PR) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With Stable Disease (SD) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Area Under the Plasma Concentration-Time Curve from Hour Zero to Hour 24 AUC[0-24] for ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Maximum Observed Plasma Concentration (Cmax) for ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Inhibition of Yes-associated Protein (YAP1) Expression [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With CR [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With PR [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Percentage of Participants With SD [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Clinical Benefit Rate (CBR) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Objective Response Rate (ORR) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Duration of Response (DoR) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Progression-free Survival (PFS) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Overall Survival (OS) [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Area Under the Plasma Concentration-Time Curve from Hour Zero to Hour 24 AUC[0-24] for ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Maximum Observed Plasma Concentration (Cmax) for ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Time to Reach the Maximum Plasma Concentration (Tmax) for ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]
- Elimination Half-Life (t1/2) of ION537 [ Time Frame: Up to disease progression, death, start of new anticancer treatment, withdrawal of consent to study participation or end of the study, whichever occurs first (up to approximately 24 weeks for analysis) ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males and females ≥ 18 years old
- Participants must have histological diagnosis of local advanced (primary or recurrent) or metastatic solid tumors that are not amenable for treatment with curative intent
- Participants must be in need of systemic treatment for their cancer and either are refractory to or have failed treatment with, are intolerant to or have refused, or are not otherwise a candidate, in the opinion of the Investigator, for any of the currently available established therapies
- Participants must have available a fresh or recent tumor tissue sample from a diagnostic biopsy/surgery or a metastatic tumor biopsy;
- Participants must have measurable disease by response evaluation criteria in solid tumors (RECIST) v1.1; or participants may have bone metastatic disease evaluable by Prostate Cancer Working Group 2 (PCWG2) for participants with metastatic castration-resistant prostate cancer (mCRPC), or according to the tumor evaluation criteria best suited and accepted for the tumor type being evaluated
- Participants must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1
- Participants must be willing and able to comply with the scheduled visits, treatment plan, laboratory tests and other specified study procedures
Exclusion Criteria:
- Known history or positive test for human immunodeficiency virus (HIV), hepatitis C virus (HCV) or chronic hepatitis B virus (HBV) infection.
- Active infection requiring intravenous (IV) antibiotics
- History of cerebrovascular accident (CVA), myocardial infarction or unstable angina within the previous 6 months before starting therapy.
- Participants with uncontrolled Type I or II diabetes mellitus (DM); uncontrolled DM
- Participants with prior anti-cancer therapy within 2 weeks prior to study enrollment or prior radiation therapy within 2 weeks prior to study enrollment.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04659096
United States, Texas | |
The University of Texas MD Anderson Cancer Center | |
Houston, Texas, United States, 77030 |
Responsible Party: | Ionis Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT04659096 |
Other Study ID Numbers: |
ION537-CS1 |
First Posted: | December 9, 2020 Key Record Dates |
Last Update Posted: | December 21, 2022 |
Last Verified: | December 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
Solid tumors Advanced solid tumors |
Neoplasms |