Sapanisertib for Neuroendocrine Tumors

Phase-Based Estimates
1
Effectiveness
2
Safety
Mayo Clinic in Arizona, Scottsdale, AZ
Neuroendocrine Tumors+7 More
Sapanisertib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Neuroendocrine Tumors

Study Summary

This study is evaluating whether a drug called sapanisertib can stop the growth of pancreatic neuroendocrine tumor cells.

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Eligible Conditions

  • Neuroendocrine Tumors
  • Cancer
  • Islet Cell Tumor
  • Carcinoma, Islet Cell
  • Carcinoma, Neuroendocrine
  • Neoplasms
  • Adenoma, Islet Cell
  • Pancreatic Neuroendocrine Tumor G1
  • Refractory Pancreatic Neuroendocrine Carcinoma
  • Pancreatic Neuroendocrine Tumor G2

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Sapanisertib will improve 2 primary outcomes and 7 secondary outcomes in patients with Neuroendocrine Tumors. Measurement will happen over the course of Assessed every 3 months for the first 2 years, every 6 months for the 3rd year, then annually up to 5 years.

Year 5
Disease Control Rate
Duration of Response
Progression-free Survival (PFS)
Year 5
Overall Response Rate
Up to 5 years
Disease control rate
Duration of response
Incidence of adverse events
Objective tumor response (complete response + partial response) as evaluated by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1
Progression-free survival (PFS)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

2 Treatment Groups

No Control Group
Treatment (sapanisertib)

This trial requires 13 total participants across 2 different treatment groups

This trial involves 2 different treatments. Sapanisertib is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Treatment (sapanisertib)
Drug
Patients receive sapanisertib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Arm A (MLN0128)
Drug
Patients receive MLN0128 orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
MLN0128
2016
Completed Phase 2
~550
Sapanisertib
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 5 years for reporting.

Closest Location

Mayo Clinic in Arizona - Scottsdale, AZ

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Refractory disease to treatment with an mTOR inhibitor Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements, are ineligible
Patients must not have poorly differentiated neuroendocrine carcinoma, high-grade neuroendocrine carcinoma, adenocarcinoid, goblet cell carcinoid and small cell carcinoma
Patients must have measurable disease
Documented radiological evidence for disease progression (measurable or nonmeasurable) =< 12 months prior to enrollment
NOTE: If patient has had previous radiation to the marker lesion(s), there must be evidence of progression since the radiation; at least one measurable lesion as per Response Evaluation Criteria in Solid Tumors (RECIST)
Prior or concurrent therapy with somatostatin analogue (SSA) is permitted; a stable dose at least 2 months prior to study start and must continue on the stable dose while receiving study treatment; SSA is not considered as systemic treatment
You have had no previous adverse events due to agents administered previously. show original
You are undergoing chemotherapy show original
Patients must have unresectable or metastatic, histologically confirmed low or intermediate grade (Klimstra Criteria) pancreatic neuroendocrine tumor (PNET) with radiological evidence of disease progression since last treatment. show original
You have a disease that is currently not amenable to surgery, radiation, or combined modality therapy with curative intent. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of neuroendocrine tumors?

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The signs of neuroendocrine neoplasia include Cushing's or Nelson's syndrome and Cushing syndrome. Abdominal bloating, loss of appetite, weight loss, peripheral edema and edema of the hands or feet are the cardinal symptoms of cirrhosis.\n

Unverified Answer

How many people get neuroendocrine tumors a year in the United States?

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Patients with neuroendocrine tumors in the United States tend to be younger than those diagnosed in other countries. Patients with neuroendocrine tumors appear to have a higher prevalence of nonfunctional tumors and lower prevalence of hyperfunctioning tumors. Patients with neuroendocrine tumors tended to have more aggressive diagnoses and higher recurrence rates.

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Can neuroendocrine tumors be cured?

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Local and whole-organ recurrence rate is very high after surgical resection for NET. The role of imaging and surgical methods is not clearly defined. Most studies have revealed that the overall survival of patients with NET is not different when compared with that without NET.

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What are common treatments for neuroendocrine tumors?

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Symptoms are managed with symptomatic drugs (selective serotonin receptor-3 agonists, selective serotonin reuptake inhibitors, and antiandrogens) or nonselective drugs (procedural sedation or tricyclic antidepressant augmentation) to reduce high levels of circulating or tissue serotonin or by blocking 5-HT3R with ondansetron alone or compounded with 5-HT antagonists. Treatments are typically given in combination. Other pharmacologic treatments used for neuroendocrine tumors include 5-HT3R antagonist ondansetron alone or combined with 5-HT antagonists, and/or opioid agonists and antagonists. The treatment of choice for the majority of cases remains undefined.

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What causes neuroendocrine tumors?

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Neuroendocrine tumors are caused by factors as diverse as genetic mutations, diet, hormones, radiation, and infections. The most common location for neuroendocrine tumors is the gastrointestinal tract. There is not a single cause.

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What is neuroendocrine tumors?

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Neuroendocrine tumors are cancerous (cancerous) tumors which usually originate in one of the endocrine glands, particularly the pancreas, that produce hormones (such as insulin or growth hormone). They are often called "islet cell tumors". Most cases of neuroendocrine tumors are associated with a condition called acromegaly. Patients with neuroendocrine tumors most often develop symptoms associated with this condition.\n

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Is sapanisertib safe for people?

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Considering the magnitude of the anticipated toxicities of sapanisertib, patients who are elderly, have a poor renal function or have a history of renal impairment may be at risks for sapanisertib-related adverse events. The risks for most other commonly expected adverse events are expected to be low.

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What is the latest research for neuroendocrine tumors?

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There is currently very limited evidence supporting the use of neuroendocrine tumors with [SURGIC] if the patient has a good performance status(<80) and cannot get chemotherapy.

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Have there been other clinical trials involving sapanisertib?

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For a better understanding concerning the efficacy and safety as well as the optimal dosage and scheduling of sapanisertib, a large, ongoing, multicentre trial is planned.

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Has sapanisertib proven to be more effective than a placebo?

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Results from a recent paper demonstrated the superiority of pan-HER inhibitor sapanisertib (dasatinib) over a placebo in patients with advanced/metastatic HER2-overexpressing breast or GI carcinomas. Because no other TKIs were studied in this population, we conclude that other targeted approaches may result in greater efficacy in HER2+ breast or gastro-oesophageal carcinoma.

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Does sapanisertib improve quality of life for those with neuroendocrine tumors?

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In a retrospective review, sapanisertib significantly improved QOL for patients with NETs as measured by PSAV, the EORTC QOL questionnaire, and patient-reported distress. In a recent study, findings suggest that the effect of sapanisertib is not limited to tumor shrinkage and should be considered when evaluating the efficacy of new anticancer drugs.

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Is sapanisertib typically used in combination with any other treatments?

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According to current USP guidelines, sapanisertib is sometimes used for its monotherapeutic properties and sometimes as part of a combination treatment with other systemic therapies or with radiation, as an alternative to newer treatments, such as the ALK or KRAS inhibitors crizotinib and osimertinib. Although this information is very valuable for patients with Ewing's sarcoma wishing to choose potential therapeutic agents for their disease, an evidence-based approach for the optimal use of sapanisertib would be more helpful to patients with other indications.

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