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NCT03008369

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

Completed Phase 2 Results posted Last updated 20 February 2024
What this trial tests

Phase 2 trial testing Laboratory Biomarker Analysis in Malignant Adrenal Gland Pheochromocytoma in 3 participants. Completed in 28 September 2022.

Timeline
31 May 2017
Primary endpoint
1 December 2020
28 September 2022

Quick facts

Lead sponsorMayo Clinic
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment3
Start date31 May 2017
Primary completion1 December 2020
Estimated completion28 September 2022
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Mayo Clinic

Who can join

18 and older, any sex, with Malignant Adrenal Gland Pheochromocytoma or Malignant Paraganglioma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Confirmed Tumor Response Rate Primary · Monthly, up to 17 months.

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

GroupValue95% CI
Treatment (Lenvatinib)1.0
Duration of Tumor Response Secondary · Every month until off treatment, at off treatment, every 3 months until PD, at PD, every 6 months after PD up to 17 months

Will be estimated using the Kaplan-Meier method.

GroupValue95% CI
Treatment (Lenvatinib)9.79.7 – NA
Patients Evaluable for Incidence of Adverse Events Assessed by Common Terminology Criteria for Adverse Events Version 4.0 Secondary · Monthly, up to 17 months.

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.

GroupValue95% CI
Treatment (Lenvatinib)3
Overall Survival Time Secondary · Every month until off treatment, at off treatment, every 3 months until PD, at PD, every 6 months after PD up to 17 months

Will be estimated using the Kaplan-Meier method.

GroupValue95% CI
Treatment (Lenvatinib)NANA – NA
Progression-free Survival Secondary · Every month until off treatment, at off treatment, every 3 months until PD, at PD or up to 17 months

Will be estimated using the Kaplan-Meier method.

GroupValue95% CI
Treatment (Lenvatinib)11.55.5 – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: Monthly, up to 17 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment (Lenvatinib)
Serious: 1/3 (33%)
Deaths: 0/3

Serious adverse events (1 terms)

ReactionSystemTreatment (Lenvatinib)
Gastrointestinal disorders - Oth specGastrointestinal disorders
Other adverse events (20 terms — click to expand)

ReactionSystemTreatment (Lenvatinib)
FatigueGeneral disorders
AnorexiaMetabolism and nutrition disorders
ProteinuriaRenal and urinary disorders
HyperthyroidismEndocrine disorders
DiarrheaGastrointestinal disorders
NauseaGastrointestinal disorders
Dry skinSkin and subcutaneous tissue disorders
HypertensionVascular disorders
HypothyroidismEndocrine disorders
ConstipationGastrointestinal disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
Mucositis oralGastrointestinal disorders
VomitingGastrointestinal disorders
Aspartate aminotransferase increasedInvestigations
Lymphocyte count decreasedInvestigations
Weight lossInvestigations
ArthralgiaMusculoskeletal and connective tissue disorders
Musculoskeletal, conn tissue - Oth specMusculoskeletal and connective tissue disorders
AnxietyPsychiatric disorders
Palmar-plantar erythrodysesthesia syndrmSkin and subcutaneous tissue disorders

Most-reported serious reactions: Gastrointestinal disorders - Oth spec.

Data from ClinicalTrials.gov NCT03008369 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Personalized Management of Pheochromocytoma and Paraganglioma.
    Nölting S, Bechmann N, Taieb D, Beuschlein F, et al · · 2022 · cited 281× · PMID 34147030 · DOI 10.1210/endrev/bnab019
  2. Current Management of Pheochromocytoma/Paraganglioma: A Guide for the Practicing Clinician in the Era of Precision Medicine.
    Nölting S, Ullrich M, Pietzsch J, Ziegler CG, et al · · 2019 · cited 112× · PMID 31597347 · DOI 10.3390/cancers11101505
  3. Paraganglioma of the Head and Neck: A Review.
    Sandow L, Thawani R, Kim MS, Heinrich MC. · · 2023 · cited 62× · PMID 36252779 · DOI 10.1016/j.eprac.2022.10.002
  4. Emerging Treatments for Advanced/Metastatic Pheochromocytoma and Paraganglioma.
    Ilanchezhian M, Jha A, Jha A, Pacak K, et al · · 2020 · cited 46× · PMID 32862332 · DOI 10.1007/s11864-020-00787-z
  5. Targeted Therapies in Pheochromocytoma and Paraganglioma.
    Wang K, Crona J, Beuschlein F, Grossman AB, et al · · 2022 · cited 45× · PMID 35973976 · DOI 10.1210/clinem/dgac471
  6. Pheochromocytomas and Paragangliomas: From Genetic Diversity to Targeted Therapies.
    Pang Y, Liu Y, Pacak K, Yang C. · · 2019 · cited 35× · PMID 30925729 · DOI 10.3390/cancers11040436
  7. The VHL/HIF Axis in the Development and Treatment of Pheochromocytoma/Paraganglioma.
    Peng S, Zhang J, Tan X, Huang Y, et al · · 2020 · cited 31× · PMID 33329393 · DOI 10.3389/fendo.2020.586857
  8. Successful Second-Line Metronomic Temozolomide in Metastatic Paraganglioma: Case Reports and Review of the Literature.
    Tena I, Gupta G, Tajahuerce M, Benavent M, et al · · 2018 · cited 25× · PMID 29720885 · DOI 10.1177/1179554918763367

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