Trial Outcomes & Findings for Lenvatinib in Treating Patients With Metastatic or Advanced Pheochromocytoma or Paraganglioma That Cannot Be Removed by Surgery (NCT NCT03008369)

Last Updated: 2024-02-20

Results Overview

Will be defined as 100% times the number of eligible patients who has started lenvatinib and whose objective tumor status was a complete response or partial response on 2 consecutive evaluations at least 4 weeks apart (using Response Evaluation Criteria in Solid Tumors version 1.1 criteria) divided by the number of eligible patients who has started lenvatinib. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Monthly, up to 17 months.

Results posted on

2024-02-20

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Lenvatinib) Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Lenvatinib in Treating Patients With Metastatic or Advanced Pheochromocytoma or Paraganglioma That Cannot Be Removed by Surgery

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Lenvatinib: Given PO Quality-of-Life Assessment: Ancillary studies
Age, Continuous	50 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Monthly, up to 17 months.

Outcome measures

Outcome measures
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Confirmed Tumor Response Rate	1.0 participants

SECONDARY outcome

Timeframe: Every month until off treatment, at off treatment, every 3 months until PD, at PD, every 6 months after PD up to 17 months

Population: All treated patients

Will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Duration of Tumor Response	9.7 Months Interval 9.7 to Upper limit not reached due to insufficient number of participants with events

SECONDARY outcome

Timeframe: Monthly, up to 17 months.

Population: All treated patients

Adverse Events are fully reported in the adverse event section of the results. All adverse events will be graded. For each type of adverse event classified as either possibly, probably, or definitely related to study treatment, the proportion of patients experiencing a severe (grade 3 or higher) adverse event will be noted per cycle. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine adverse event patterns.

Outcome measures

Outcome measures
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Patients Evaluable for Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0	3 Participants

SECONDARY outcome

Timeframe: Every month until off treatment, at off treatment, every 3 months until PD, at PD, every 6 months after PD up to 17 months

Population: All treated patients

Will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Overall Survival Time	NA Months Kaplan-Meier median, lower limit, and upper limit not reached due to insufficient number of participants with events.

SECONDARY outcome

Timeframe: Every month until off treatment, at off treatment, every 3 months until PD, at PD or up to 17 months

Population: All treated patients

Will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	Treatment (Lenvatinib) n=3 Participants Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.
Progression-free Survival	11.5 Months Interval 5.5 to Upper limit not reached due to insufficient number of participants with events

SECONDARY outcome

Timeframe: 5 years

Population: No patients made it to the QoL timepoint or submitted QoL data after treatment.

Descriptive statistics, and scatter plots will form the basis of presentation of these data both overall and by other outcomes (toxicity, response and survival measures). Correlations between the quality of life outcomes and other outcome measures will be carried out by standard parametric and nonparametric tests (e.g. Pearson's and Spearman's rho). Comparison between continuous variables will be made with Wilcoxon rank sum tests, Fisher's exact tests will be used to determine differences between categorical variables, and Log-rank test will be used to test differences between time-to-event ou

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Wilcoxon rank sum tests will be used to examine whether fold changes in a given biomarker during the first cycle of treatment differs between whose tumor responded to treatment and those whose tumor did not. Time series plots will be constructed.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Will examine associations between tumor response and somatic mutational status in archived tumors, or germline mutational status in patient's peripheral blood mononuclear cells, (presence of SDHD, SDHB, RET, VHL, neurofibromatosis type-1).

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Lenvatinib)

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Lenvatinib) n=3 participants at risk Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Lenvatinib: Given PO Quality-of-Life Assessment: Ancillary studies
Gastrointestinal disorders Gastrointestinal disorders - Oth spec	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0

Other adverse events

Other adverse events
Measure	Treatment (Lenvatinib) n=3 participants at risk Patients receive lenvatinib PO once daily on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Lenvatinib: Given PO Quality-of-Life Assessment: Ancillary studies
Endocrine disorders Hyperthyroidism	66.7% 2/3 • Number of events 4 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Endocrine disorders Hypothyroidism	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Constipation	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Diarrhea	66.7% 2/3 • Number of events 12 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Gastroesophageal reflux disease	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Mucositis oral	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Nausea	66.7% 2/3 • Number of events 11 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Gastrointestinal disorders Vomiting	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
General disorders Fatigue	100.0% 3/3 • Number of events 19 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Investigations Aspartate aminotransferase increased	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Investigations Lymphocyte count decreased	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Investigations Weight loss	33.3% 1/3 • Number of events 2 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Metabolism and nutrition disorders Anorexia	100.0% 3/3 • Number of events 5 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Musculoskeletal and connective tissue disorders Arthralgia	33.3% 1/3 • Number of events 1 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Musculoskeletal and connective tissue disorders Musculoskeletal, conn tissue - Oth spec	33.3% 1/3 • Number of events 2 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Psychiatric disorders Anxiety	33.3% 1/3 • Number of events 4 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Renal and urinary disorders Proteinuria	100.0% 3/3 • Number of events 15 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Skin and subcutaneous tissue disorders Dry skin	66.7% 2/3 • Number of events 4 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrm	33.3% 1/3 • Number of events 4 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0
Vascular disorders Hypertension	66.7% 2/3 • Number of events 17 • Monthly, up to 17 months. Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0

Additional Information

Ashish Vitthalrao Chintakuntlawar, MBBS, Ph.D.

Mayo Clinic

Phone: 5072930504

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place