Trial Outcomes & Findings for Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) (NCT NCT04195555)

NCT ID: NCT04195555

Last Updated: 2026-05-04

Results Overview

A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system tumors).

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Up to 2 years from study entry

Results posted on

2026-05-04

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Ivosidenib) Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Ivosidenib) Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Overall Study Progressive disease	1
Overall Study Refusal by patient/parent/guardian	1
Overall Study No treatment	1

Baseline Characteristics

Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Ivosidenib) n=3 Participants Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Age, Continuous	16.7 years STANDARD_DEVIATION 2.5 • n=54 Participants
Sex: Female, Male Female	1 Participants n=54 Participants
Sex: Female, Male Male	2 Participants n=54 Participants
Age, Categorical <=18 years	2 Participants n=54 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=54 Participants
Age, Categorical >=65 years	0 Participants n=54 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=54 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=54 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=54 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=54 Participants
Race (NIH/OMB) Asian	1 Participants n=54 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=54 Participants
Race (NIH/OMB) Black or African American	0 Participants n=54 Participants
Race (NIH/OMB) White	2 Participants n=54 Participants
Race (NIH/OMB) More than one race	0 Participants n=54 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=54 Participants
Region of Enrollment United States	3 participants n=54 Participants

PRIMARY outcome

Timeframe: Up to 2 years from study entry

Population: One patient did not receive any protocol therapy and is excluded from this analysis.

Outcome measures

Outcome measures
Measure	Treatment (Ivosidenib) n=2 Participants Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Objective Response Rate (ORR; Complete Response + Partial Response) in Pediatric Patients Treated With Ivosidenib	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Up to 6 months from study entry

The Kaplan-Meier method will be used to estimate the 6 month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years from study entry

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-dose, 1, 2, 3, 4, and 6-8 hours after dose on cycle 1, day 1; pre-dose on cycle 1, day 2; pre-dose, 1, 2, 3, 4, and 6-8 hours after dose on cycle 1, day 15; and pre-dose on cycle 2, day 1 and cycle 4, day 1

A descriptive analysis of PK parameters will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).

Outcome measures

Outcome data not reported

SECONDARY outcome

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Will evaluate other biomarkers as predictors of response to ivosidenib and whether tumors that harbor different missense mutations or fusions will demonstrate differential response to treatment. A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 5 years

Will explore approaches to the profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid. A descriptive analysis will be performed and will be summarized with simple summary statistics.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Ivosidenib)

Serious events: 1 serious events

Other events: 2 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Ivosidenib) n=2 participants at risk Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.

Other adverse events

Other adverse events
Measure	Treatment (Ivosidenib) n=2 participants at risk Patients receive ivosidenib PO QD. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders Nausea	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Gastrointestinal disorders Vomiting	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
General disorders Fatigue	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Investigations Blood bicarbonate decreased	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Investigations Lymphocyte count decreased	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Investigations Neutrophil count decreased	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Investigations White blood cell decreased	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Metabolism and nutrition disorders Hyperphosphatemia	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Nervous system disorders Headache	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Nervous system disorders Seizure	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
Skin and subcutaneous tissue disorders Rash acneiform	50.0% 1/2 • Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: (626) 241-1500

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60