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Comparing Retreatment of 177Lu-DOTATATE PRRT Versus Everolimus in Patients With Metastatic Unresectable Midgut Neuroendocrine Tumors, NET RETREAT Trial

Phase 2
Recruiting
Conditions
Metastatic Midgut Neuroendocrine Tumor G1
Metastatic Midgut Neuroendocrine Tumor
Metastatic Midgut Neuroendocrine Tumor G2
Unresectable Midgut Neuroendocrine Tumor
Interventions
Procedure: Biospecimen Collection
Procedure: Computed Tomography
Other: Quality-of-Life Assessment
Other: Questionnaire Administration
Registration Number
NCT05773274
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

This phase II trial compares the effect of retreatment with 177Lu-DOTATATE peptide receptor radionuclide therapy (PRRT) to the usual approach of treatment with everolimus in patients who have previously received 177Lu-DOTATATE for midgut neuroendocrine tumor (NET) that has spread from where it first started (primary site) to other places in the body (metastatic) and that cannot be removed by surgery (unresectable). PRRT is a type of radiation therapy for which a radioactive chemical is linked to a peptide (small protein) that targets cancer cells. When this radioactive peptide is injected into the body, it binds to a specific receptor found on some cancer cells. The radioactive peptide builds up in these cells and helps kill the cancer cells without harming normal cells. In this trial 177Lu-DOTATATE is used for PRRT. 177Lu-DOTATATE PRRT may increase the length of time until worsening of the midgut NET compared to the usual approach. Everolimus is in a class of medications called kinase inhibitors. It is also a type of angiogenesis inhibitor. Everolimus works by stopping cancer cells from reproducing and by decreasing blood supply to the cancer cells. Retreating with 177Lu-DOTATATE may work better than everolimus in shrinking or stabilizing tumor in patients with metastatic and unresectable NET who were previously treated with 177Lu-DOTATATE.

Detailed Description

PRIMARY OBJECTIVE:

I. To evaluate the effect of lutetium Lu 177 dotatate (177Lu-DOTATATE) versus (vs.) everolimus on progression-free survival (PFS) in patients with metastatic/unresectable midgut neuroendocrine tumour (NET) who have progressed following previous peptide receptor radionuclide therapy (PRRT).

SECONDARY OBJECTIVES:

I. To evaluate the toxicity and safety of 177Lu-DOTATATE and everolimus. II. To determine the effect of 177Lu-DOTATATE vs. everolimus on overall response rate (ORR).

III. To evaluate the effect of 177Lu-DOTATATE vs. everolimus on overall survival (OS).

IV. To evaluate post progression survival (PPS) and time to second objective disease progression (PFS2) for patients randomized to Arm 2 of the study and crossed over to Arm 1 at time of objective progression per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.

V. To evaluate the effect of 177Lu-DOTATATE vs. everolimus on patient quality of life (QoL).

OUTLINE: Patients are randomized to 1 of 2 arms.

ARM I: Patients receive 177Lu-DOTATATE intravenously (IV) every 8 weeks (Q8W). Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo computed tomography (CT) scan and collection of blood samples while on study.

ARM II: Patients receive everolimus orally (PO) on a daily basis (QD). Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
100
Inclusion Criteria
  • Patients must be at least >= 18 years of age

  • Metastatic, histologically confirmed grade 1 or 2 well-differentiated midgut neuroendocrine tumours, including NETs of unknown primary thought to be of midgut origin, with positive Gallium-68 DOTATATE scan or Copper-64 DOTATATE scan within the last 12 months is recommended but within the last 36 months is allowed. Lesions on Gallium-68 or Copper-64 DOTATATE scan will be considered positive if the maximum standardized uptake value (SUVmax) of target lesion is > SUV mean of normal liver parenchyma

  • Have received 3 or 4 cycles of PRRT using 177Lu-DOTATATE or a cumulative exposure of 22,200 MBq (600mCi) or 29,600 MBq (800 mCi) within a 52-week period. Previous therapy with everolimus for a maximum period of 1 month is permitted. No previous targeted alpha therapy is permitted. No previous alkylator therapy (i.e. Temodar) is permitted

  • Have had radiological progression per RECIST 1.1 after prior PRRT treatment and no sooner than 12 months from last scan performed post completion of initial PRRT where either stable disease, partial response, or complete response has been maintained throughout

  • Have not received any intervening therapy after initial PRRT

  • No ongoing toxicity from prior PRRT that is grade 3 or higher according to Common Terminology Criteria for Adverse Events (CTCAE) 5.0

  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2

  • Hemoglobin >= 80 g/L (>= 8.0 g/dL) (measured within 28 days prior to enrollment)

  • Absolute neutrophil count >= 1.0 x 10^9/L (>= 1000/mm^3) (measured within 28 days prior to enrollment)

  • Platelets >= 80 x 10^9/L (>= 80 x 10^3/mm^3) (measured within 28 days prior to enrollment)

  • Total bilirubin < 1.5 x upper limit of normal (ULN) (upper limit of normal) (measured within 28 days prior to enrollment)

    • If confirmed Gilbert's, eligible providing =< criteria x ULN
  • Creatinine clearance > 50 mL/min (measured within 28 days prior to enrollment)

    • Creatinine clearance to be measured directly by 24 hour urine sampling or as calculated by Cockcroft and Gault equation
  • Prior or current use of somatostatin analogues is allowed for carcinoid syndrome control or in PRRT re-treatment patient population (Arm 1). Patients randomized to everolimus (Arm 2) will not be allowed to continue somatostatin analogues unless they have functional carcinoid syndrome

  • Patient consent must be appropriately obtained in accordance with applicable local and regulatory requirements. Each patient must sign a consent form prior to enrollment in the trial to document their willingness to participate

  • Patients of childbearing potential must have agreed to use a highly effective contraceptive method during protocol treatment and for 7 months after the last dose of protocol treatment. A woman is considered to be of "childbearing potential" if she has had menses at any time in the preceding 12 consecutive months. In addition to routine contraceptive methods, "effective contraception" also includes heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal ligation, or vasectomy/vasectomized partner. However, if at any point a previously celibate patient chooses to become heterosexually active during the time period for use of contraceptive measures outlined in the protocol, he/she is responsible for beginning contraceptive measures

    • Women of childbearing potential will have a pregnancy test to determine eligibility as part of the Pre-Study Evaluation; this may include an ultrasound to rule-out pregnancy if a false-positive is suspected. For example, when beta-human chorionic gonadotropin is high and partner is vasectomized, it may be associated with tumour production of human chorionic gonadotropin (hCG), as seen with some cancers. Patient will be considered eligible if an ultrasound is negative for pregnancy
  • Patients must be accessible for treatment, response assessment, and follow up. Patients enrolled on this trial must be treated and followed at the participating center. Investigators must assure themselves the patients enrolled on this trial will be available for complete documentation of the treatment, adverse events, and follow-up

    • Patients must agree to return to their primary care facility for any adverse events which may occur through the course of the trial
  • Patient must have access to everolimus. In the event that site/investigator is unable to provide access to the drug, patient will not be eligible for this trial

  • Human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial

  • Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial

Exclusion Criteria
  • Major surgical procedures within 6 weeks from randomization date
  • Known brain metastases, unless these metastases have been treated, stabilized and off steroids for at least 4 weeks prior to enrollment in the study. Patients with a history of brain metastases must have a head CT and/or MRI with contrast to document stable disease prior to enrollment in the study
  • Uncontrolled congestive heart failure no worse than New York Heart Association Class (NYHA) IIB
  • Inability to swallow oral medications or gastrointestinal disease limiting absorption of oral agents
  • Patients with any other significant medical or surgical condition, currently uncontrolled by treatment, which may interfere with completion of the study
  • Pregnant women are excluded from this study because 177Lu-DOTATATE is a peptide receptor radionuclide therapy with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with 177Lu-DOTATATE, breastfeeding should be discontinued if the mother is treated with everolimus or 177Lu-DOTATATE and for 2.5 months following the last treatment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm I (177Lu-DOTATATE)Lutetium Lu 177 DotatatePatients receive 177Lu-DOTATATE IV Q8W. Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo CT scan and collection of blood samples while on study.
Arm II (everolimus)Computed TomographyPatients receive everolimus PO QD. Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.
Arm I (177Lu-DOTATATE)Biospecimen CollectionPatients receive 177Lu-DOTATATE IV Q8W. Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo CT scan and collection of blood samples while on study.
Arm I (177Lu-DOTATATE)Computed TomographyPatients receive 177Lu-DOTATATE IV Q8W. Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo CT scan and collection of blood samples while on study.
Arm II (everolimus)Biospecimen CollectionPatients receive everolimus PO QD. Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.
Arm I (177Lu-DOTATATE)Quality-of-Life AssessmentPatients receive 177Lu-DOTATATE IV Q8W. Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo CT scan and collection of blood samples while on study.
Arm I (177Lu-DOTATATE)Questionnaire AdministrationPatients receive 177Lu-DOTATATE IV Q8W. Treatment repeats for two cycles in the absence of disease progression or unacceptable toxicities. Patients also undergo CT scan and collection of blood samples while on study.
Arm II (everolimus)Quality-of-Life AssessmentPatients receive everolimus PO QD. Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.
Arm II (everolimus)Questionnaire AdministrationPatients receive everolimus PO QD. Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.
Arm II (everolimus)EverolimusPatients receive everolimus PO QD. Treatment continues in the absence of disease progression or unacceptable toxicities. Patients whose cancer worsens may cross over to ARM I. Patients also undergo CT scan and collection of blood samples while on study.
Primary Outcome Measures
NameTimeMethod
Progression-free survival (PFS)From randomization to any documented evidence of tumour progression or death from any cause, assessed up to 3 years

The PFS of patients of both treatment groups will be described by Kaplan-Meier method and the median PFS will be estimated using the same method. A one-sided stratified log-rank test adjusting for the stratification factor at randomization will be the primary method to compare the difference in PFS between the experimental and control treatments, at the 5% level. A stratified Cox model adjusting for the stratification factor at randomization and with one single treatment covariate will be used to estimate the hazard ratio and associated one-sided 95% confidence interval.

Secondary Outcome Measures
NameTimeMethod
Overall survival (OS)From randomization to death from any cause, assessed up to 3 years

The OS of patients of both treatment groups will be described by Kaplan-Meier method and the median OS will be estimated using the same method. A one-sided stratified log-rank test adjusting for the stratification factor at randomization will be the primary method to compare the difference in OS between the experimental and control treatments, at the 5% level. A stratified Cox model adjusting for the stratification factor at randomization and with one single treatment covariate will be used to estimate the hazard ratio and associated one-sided 95% confidence interval.

Post progression survival (PPS)From objective progression on everolimus to objective progression or death from any cause after cross-over to receive 177Lu-DOTATATE, assessed up to 3 years

Median PPS and associated two-sided 90% confidence interval will be estimated using the Kaplan-Meier method.

Objective response rate (ORR)Up to 3 years

Defined as the proportion of patients with a documented complete response and partial response based on Response Evaluation Criteria in Solid Tumors 1.1. The primary estimate of ORR will be based on all patients randomized. A Cochran-Mantel-Haenszel test adjusting for the stratification factor at the time of randomization will be used to compare the objective response rates between two arms.

Time to second objective disease progression (PFS2)From randomization to objective tumor progression or death from any cause after the cross-over, assessed up to 3 years

Median PFS2 and associated two-sided 90% confidence interval will be estimated using the Kaplan-Meier method.

Trial Locations

Locations (28)

University of Alabama at Birmingham Cancer Center

🇺🇸

Birmingham, Alabama, United States

Mayo Clinic Hospital in Arizona

🇺🇸

Phoenix, Arizona, United States

UCHealth University of Colorado Hospital

🇺🇸

Aurora, Colorado, United States

UM Sylvester Comprehensive Cancer Center at Aventura

🇺🇸

Aventura, Florida, United States

UM Sylvester Comprehensive Cancer Center at Coral Gables

🇺🇸

Coral Gables, Florida, United States

UM Sylvester Comprehensive Cancer Center at Deerfield Beach

🇺🇸

Deerfield Beach, Florida, United States

Mayo Clinic in Florida

🇺🇸

Jacksonville, Florida, United States

University of Miami Miller School of Medicine-Sylvester Cancer Center

🇺🇸

Miami, Florida, United States

UM Sylvester Comprehensive Cancer Center at Kendall

🇺🇸

Miami, Florida, United States

UM Sylvester Comprehensive Cancer Center at Plantation

🇺🇸

Plantation, Florida, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

University of Chicago Comprehensive Cancer Center

🇺🇸

Chicago, Illinois, United States

Northwestern Medicine Cancer Center Kishwaukee

🇺🇸

DeKalb, Illinois, United States

Northwestern Medicine Cancer Center Delnor

🇺🇸

Geneva, Illinois, United States

UC Comprehensive Cancer Center at Silver Cross

🇺🇸

New Lenox, Illinois, United States

University of Chicago Medicine-Orland Park

🇺🇸

Orland Park, Illinois, United States

Northwestern Medicine Cancer Center Warrenville

🇺🇸

Warrenville, Illinois, United States

University of Kentucky/Markey Cancer Center

🇺🇸

Lexington, Kentucky, United States

Henry Ford Hospital

🇺🇸

Detroit, Michigan, United States

Mayo Clinic in Rochester

🇺🇸

Rochester, Minnesota, United States

University of New Mexico Cancer Center

🇺🇸

Albuquerque, New Mexico, United States

University of Rochester

🇺🇸

Rochester, New York, United States

Ohio State University Comprehensive Cancer Center

🇺🇸

Columbus, Ohio, United States

Huntsman Cancer Institute/University of Utah

🇺🇸

Salt Lake City, Utah, United States

BCCA-Vancouver Cancer Centre

🇨🇦

Vancouver, British Columbia, Canada

CancerCare Manitoba

🇨🇦

Winnipeg, Manitoba, Canada

Ottawa Hospital and Cancer Center-General Campus

🇨🇦

Ottawa, Ontario, Canada

Odette Cancer Centre- Sunnybrook Health Sciences Centre

🇨🇦

Toronto, Ontario, Canada

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