Neoplasms

All Conditions

Rare Diseases

Pheochromocytoma

Paraganglioma

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Nerve Tissue

Paragangliomas 1

Neuroendocrine Tumor

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Lenvatinib

Protein Kinase Inhibitors

Enzyme Inhibitors

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

Quality-of-Life Assessment

Ashish Chintakuntlawar

This phase II trial studies how well lenvatinib works in treating patients with pheochromocytoma or paraganglioma that has spread to other places in the body or cannot be removed by surgery. Lenvatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

PRIMARY OBJECTIVES:

I. To determine the anti-tumor activity of lenvatinib (overall response rate; \[ORR\]) in patients with metastatic or advanced unresectable pheochromocytomas and paragangliomas.

SECONDARY OBJECTIVES:

I. To determine progression-free survival (PFS). II. To determine overall survival (OS). III. To determine duration of tumor response. IV. To determine safety and tolerability of lenvatinib. V. To assess patient reported quality of life using EuroQol Five-Dimensional Five Level Scale Questionnaire (EQ-5D-5L) and Functional Assessment of Cancer Therapy-General (FACT-G).

TERTIARY OBJECTIVES:

I. For patients with secretory tumors, to examine changes in plasma metanephrine levels and urinary catecholamine and/or metanephrine levels.

II. For patients with secretory tumors, to examine whether lenvatinib-induced changes in plasma metanephrines and urinary catecholamine and/or metanephrine levels during the first cycle of treatment may be associated with objective tumor response.

III. To examine associations between tumor response and somatic mutational status in archived tumors, or germline mutational status (presence of SDHD, SDHB, RET, VHL, neurofibromatosis type-1).

OUTLINE:

Patients receive lenvatinib orally (PO) once daily (QD) on days 1-28. Courses repeat every 28 days for up to 5 years in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 or 6 months for up to 5 years.

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

Phase II Trial of Lenvatinib in Metastatic or Advanced Pheochromocytoma and Paraganglioma

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.

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).

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

Will be estimated using the Kaplan-Meier method.

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.

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

National Cancer Institute (NCI)

Mayo Clinic

Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0

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

Treatment (Lenvatinib)

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Ashish Vitthalrao Chintakuntlawar, MBBS, Ph.D.

Confirmed Tumor Response Rate

Duration of Tumor Response

Patients Evaluable for Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0

Overall Survival Time

Progression-free Survival

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

Quality of Life Assessed by EQ-5D and FACT-G

Changes in Urinary Catecholamine and Metanephrine Levels

Germline Mutational Status in Peripheral Blood Mononuclear Cells

Somatic Mutational Status in Peripheral Blood Mononuclear Cells

Overall Study

Paraganglioma, Extra-Adrenal

Patients receive anlotinib hydrochloride 12mg orally once daily on days 1-14. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

anlotinib hydrochloride

Anli Tong

anli tong

This phase II trial studies how well anlotinib hydrochloride works in treating patients with metastatic pheochromocytoma or paraganglioma. Anlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

PRIMARY OBJECTIVES:

To determine the anti-tumor activity of anlotinib hydrochloride (Objective Response rate,ORR) in patients with metastatic pheochromocytomas or paragangliomas.

SECONDARY OBJECTIVES:

I. To assess safety profile of anlotinib. II. To assess progression-free survival time. III. To assess disease control rate.

OUTLINE:

Patients receive anlotinib hydrochloride 12mg orally once daily on days 1-14. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Patients undergo urine and blood sample collection, imaging examinations at baseline and periodically during study.

After completion of study therapy, patients are followed up every 3-6 months.

The Efficacy and Safety of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma

A Phase 2 Study of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma

PFS is defined as the time from the first day of treatment to the first documented disease progression per RECIST 1.1 criteria and the Kaplan-Meier curve. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in the RECIST criteria.

Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events version 4.0

Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)

Defined for all patients whose tumor met the criteria of CR or PR or stable disease(SD)

Peking Union Medical College Hospital

Gland and Hormone Related Diseases

Symptoms and General Pathology

Carcinoma

Thyroid Neoplasms

Thyroid Diseases

Body Weight

Endocrine Gland Neoplasms

Carcinoma, Medullary

Neoplasms, Glandular and Epithelial

Endocrine System Diseases

Carcinoma, Neuroendocrine

Adenocarcinoma

Head and Neck Neoplasms

Thyroid Cancer, Medullary

Amino Acids

Tyrosine Kinase Inhibitors

Tyrosine

Patients treated with cabozantinib s-malate or lenvatinib mesylate are observed for body weight, skeletal muscle and adipose tissue changes. Patients complete 3 to 4 questionnaires every 2 weeks for 6 months and then monthly up to 12 months. Patients also undergo physical assessments and body composition measurements by DXA and CT scans at baseline, months 3, 6, and 12.

Computed Tomography

Dual X-ray Absorptiometry

Physical Examination

Questionnaire Administration

Cabozantinib

Naifa L Busaidy

The goal of this clinical research study is to learn about possible weight, muscle, and/or fat loss in patients receiving cabozantinib or lenvatinib.

Baseline Visit

If you agree to take part in this study, you will have the following tests and procedures at a baseline visit within 10 days after starting either cabozantinib or lenvatinib:

Your height, weight, waist, and hips will be measured. You will have basic tests of your physical strength and balance. Your hand grip strength will be measured, you will be asked to rise from a chair without using your arms to push off, and you will complete a 6-minute walk test.

You will have a full-body dual-energy x-ray absorptiometry (DXA) scan to measure the amount of fat and muscle in your body. Images from a standard computed tomography (CT) scan will also be collected and reviewed for this purpose. If you are scheduled only for a chest CT scan as part of your regular care, your abdomen will also be scanned at the same time for research purposes.

You will meet with a dietician to talk about your current and past eating habits.

You will fill out questionnaires about any pain or other symptoms you may have had; about any gastrointestinal (GI) side effects you may be having before you start chemotherapy; about the amount of physical exercise and activity you get; and about your food intake over the last 24 hours. Together the questionnaires should take about 20-25 minutes to complete.

You will be receiving cabozantinib or lenvatinib as part of your standard of care.

Study Visits After the baseline visit, you will have study visits on the following schedule. Some of these visits will be at the same time as your scheduled clinic visits with your regular doctor. However, some assessments will be done in between clinic visits online or by phone.

Month 1 and 2:

One (1) time every 2 weeks (+/- 1 week) during Months 1 and 2, you will fill out 3 questionnaires at home:

You will be asked about any gastrointestinal (GI) and other side effects you may be having. This will be done by phone and should take about 5-10 minutes.

You will fill out an online questionnaire at home about your eating habits over the last 24 hours. If you do not have internet access, you will complete the questionnaire over the phone with a member of the study staff. This should take about 10 minutes.

You will complete a questionnaire about any symptoms you may be having online or by phone. This should take about 5-8 minutes.

At Months 3, 6, and 12 (± 4 weeks) you will come to the clinic:

Your height, weight, waist, and hips will be measured. You will have the same tests of your physical strength and balance that you had at the baseline visit.

You will complete the same questionnaires you completed at the baseline visit. These will be done on paper or on a computer or tablet.

You will have a DXA scan to measure the amount of fat and muscle in your body. If you are having certain GI side effects (such as diarrhea, nausea, vomiting, mouth sores, and so on), the study doctor will talk with you about receiving standard drugs to help with those side effects. You may ask the study staff for information about how the drugs are given and their risks. You may also meet with a dietician, who may recommend ways to help improve some side effects.

Between clinic visits:

Two (2) times per month (+/- 1 week) between Months 3 and 6, and once a month (+/- 1 week) between Months 6 and 12, you will complete 3 questionnaires at home about your food intake over the past 24 hours; any symptoms you may be having; and any GI or other side effects you may be having. The questionnaires may be completed online or by phone and together should take about 20 minutes.

Length of Study Participation Your participation in this study will be over after the Month 12 visit

Effect of Cabozantinib S-Malate or Lenvatinib Mesylate on Weight and Body Composition in Patients With Metastatic Endocrine Cancer

Effects of Tyrosine Kinase Inhibitors on Body Composition in Endocrine Tumors -- A Pilot Study

Summary descriptive statistics, including proportions, medians, means, 95% confidence intervals, and other simple statistics will be provided for demographics, tumor response rates and blood markers. 95% CI will be reported for the accuracy of DXA compared to CT. The correlation between change of weight/body composition and tumor response will be investigated by logistic regression.

2-sided paired t-test will be used to measure weight change after successful intervention of diarrhea and anorexia. Linear mixed-effects model will be used to explore the longitudinal weight changes. Summary descriptive statistics, including proportions, medians, means, 95% confidence intervals, and other simple statistics will be provided for demographics, gastrointestinal adverse events, body composition changes and blood markers. 95% confidence intervals (CI) will be reported for the accuracy of DXA compared to computed tomography CT. The correlation between change of weight/body composition and tumor response will be investigated by logistic regression.

Summary descriptive statistics, including proportions, medians, means, 95% confidence intervals, and other simple statistics will be provided for demographics, body composition changes and blood markers. 95% CI will be reported for the accuracy of DXA compared to CT. The correlation between change of weight/body composition and tumor response will be investigated by logistic regression.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Lenvatinib in Treating Patients With Metastatic or Advanced Pheochromocytoma or Paraganglioma That Cannot Be Removed by Surgery	NCT03008369	Malignant Adren... Malignant Parag... Metastatic Adre...	Laboratory Biom... Lenvatinib Quality-of-Life...	18 Years -	Mayo Clinic
The Efficacy and Safety of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma	NCT04860700	Malignant Adren... Malignant Parag... Pheochromocytom... Paraganglioma, ... Paraganglioma, ...	anlotinib hydro...	18 Years - 75 Years	Peking Union Medical College Hospital
Effect of Cabozantinib S-Malate or Lenvatinib Mesylate on Weight and Body Composition in Patients With Metastatic Endocrine Cancer	NCT02592356	Differentiated ... Malignant Adren... Malignant Parag... Thyroid Gland M...	Computed Tomogr... Dual X-ray Abso... Physical Examin... Quality-of-Life... Questionnaire A... Cabozantinib Lenvatinib	18 Years -	M.D. Anderson Cancer Center

Spots Global Cancer Trial Database for malignant adrenal gland pheochromocytoma