Study of Tamoxifen in Well Differentiated Neuroendocrine Tumors and Hormone Receptor Positive Expression

NCT03870399 | Completed Phase 2 FDA Drug

• 1 other identifier: 2626/18
• Study Type: interventional
• Target: 23
• Locations: 2 countries
• Sites: 2

Brief Summary

This is a single-arm, unicentric, single-stage clinical study of tamoxifen for patients with well differentiated neuroendocrine tumors and radiological progression with positive (\> 1 percent) HR (estrogen and / or progesterone) expression by IHC. It will evaluate if Tamoxifen exerts antitumor action in patients with well differentiated NET and positive for the expression of HR, estrogen and / or progesterone.

Conditions

Neuroendocrine Tumors; Progesterone Receptor Positive Tumor; Estrogen Receptor Positive Tumor

Keywords

neuroendocrine tumor; tamoxifen; Progesterone Receptor; Estrogen Receptor

Outcome Measures

Primary Outcomes (1)

• Disease control rate

  Defined by absence of radiological progression in conventional imaging examinations by RECIST 1.1. Isolated increase of biomarker (chromogranin A) or specific hormone will not be considered progression.

  at 24 weeks after initiation of tamoxifen (at the end of cycle 6 - each cycle is 28 days)

Secondary Outcomes (5)

• Progression-free survival

  Through study completion, an average of 5 years

• Rate of Biochemical response

  Through study completion, an average of 5 years

• Radiological response rate

  Through study completion, an average of 5 years

• Disease control rate

  Through study completion, an average of 5 years

• Incidence of Treatment-related Adverse Events

  Through study completion, an average of 5 years

Other Outcomes (3)

• PET-CT gallium-68 intake variation

  Through study completion, an average of 3 years

• PET-CT gallium-68 number variation

  Through study completion, an average of 3 years

• CTC positivity rate

  Through study completion, an average of 3 years

Study Arms (1)

Tamoxifen

EXPERIMENTAL

The participants will receive tamoxifen 20mg orally once daily with a glass of water. Each cycle will be defined for 42 days (6 weeks).

Drug: Tamoxifen

Interventions

Tamoxifen DRUG

The treatment to be used will be tamoxifen 20mg orally once daily.

Tamoxifen

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age greater than or equal to 18 years
• Histological diagnosis of well differentiated NET (typical and atypical lung carcinoids, NET G1, NET G2 of all gastroenteropancreatic sites and pancreatic NET G3 according to WHO 2017 classification) 20 advanced / metastatic, inoperable, with no possibility of curative treatment
• Immunohistochemical expression ≥ 1 percent for estrogen and / or progesterone receptor
• Disease with radiological progression (at least 10 percent tumor volume growth) in the last 12 months before day 1 cycle 1.
• No possibility of established treatments due to lack of access, risk of toxicities or without clinical indication. Patients who meet criteria for watchful waiting (low-dose disease and non-functioning NET) may be included.
• Measurable disease
• ECOG performance scale 0 to 2.
• Adequate organic function as defined by the following criteria:
• serum aspartate aminotransferase (AST), serum alanine aminotransferase (ALT) ≤ 2.5 times the upper limit of local laboratory normality (LSN-LL);
• Total serum bilirubin ≤ 2.0 x ULN-LL;
• Absolute neutrophil count ≥ 1,500 / mm\^3;
• Platelet count ≥ 80,000 / mm\^3;
• Hemoglobin ≥ 9.0 g / dL;
• Estimated creatinine clearance by the MDRD equation ≥ 30ml / min
• Albumin ≥ 3.5 g / dL;

You may not qualify if:

• Patients already on tamoxifen, but other prior treatment are allowed
• Patients with aggressive disease requiring cytotoxic therapy or locoregional therapies (eg hepatic embolization)
• A history of serious clinical or psychiatric illness that, by clinical judgment, may involve participation risk in this study
• Patients participating in other protocols with experimental drugs.
• Patients with oral food difficulties.
• Patients who underwent major recent surgery less than 4 weeks previously.
• Patients receiving chemotherapy or other oncologic therapy for less than 3 weeks.
• Patients who use oral anticoagulation
• Previous history of deep vein thrombosis or pulmonary embolism in the last 12 months.
• Pregnant or lactating patients.
• Patients with postmenopausal vaginal bleeding with no defined etiology.
• Patients with breast cancer who need to use tamoxifen for this neoplasm
• Another synchronous neoplasm that requires systemic treatment

Sponsors & Collaborators

• AC Camargo Cancer Center: lead
• H. Lee Moffitt Cancer Center and Research Institute: collaborator

Study Sites (2)

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612, United States

Location

AC Camargo Cancer Center

São Paulo, São Paulo, 01525000, Brazil

Location

Related Publications (22)

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  PMID: 37529158 DERIVED

MeSH Terms

Conditions

Neuroendocrine Tumors

Interventions

Tamoxifen

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Stilbenes; Benzylidene Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals

Study Officials

• Rachel SP Riechelmann, Phd

  Fundacao Antonio Prudente

  PRINCIPAL INVESTIGATOR

• Jonathan R Strosberg, MD

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Clinical Oncology Department

Model Details: A single-arm, unicentric, single-stage clinical study

Study Record Dates

• First Submitted: March 6, 2019
• First Posted: March 12, 2019
• Study Start: March 13, 2019
• Primary Completion: May 13, 2023
• Study Completion: May 13, 2023
• Last Updated: May 6, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); BR (1)