Last reviewed · How we verify

NCT02420691

Ribociclib in Treating Patients With Advanced Neuroendocrine Tumors of Foregut Origin

Completed Phase 2 Results posted Last updated 30 October 2020
What this trial tests

Phase 2 trial testing Laboratory Biomarker Analysis in Advanced Digestive System Neuroendocrine Neoplasm in 21 participants. Completed in 5 June 2019.

Timeline
25 August 2015
Primary endpoint
5 June 2019
5 June 2019

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment21
Start date25 August 2015
Primary completion5 June 2019
Estimated completion5 June 2019
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

18 and older, any sex, with Advanced Digestive System Neuroendocrine Neoplasm or Duodenal Neuroendocrine Tumor G1. 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.

Number of Participants With Response Rate Per Response Evaluation Criteria in Solid Tumors Version 1.1 Primary · 3 years 10 months

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: 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.

GroupValue95% CI
Study Participants19
Number of Participants With Clinical Benefit Rate Secondary · At 6 months

Number of Participants that did not have progressive disease at 6 months.

GroupValue95% CI
Study Participants19
Progression Free Survival (PFS) Secondary · From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 24 months

PFS is the length of time during and after the treatment that a participant lives with the disease but it does not get worse.The Kaplan-Meier (KM) method will be used to estimate the PFS.

GroupValue95% CI
Study Participants10.447.37 – 13.49
Change in pRB With Treatment Secondary · Baseline (Pre-Treatment) and Cycle 2 Day 1, each Cycle is 28 days

Decrease in pRB as Measured by Immunohistochemistry (IHC) in Biopsies From Baseline and From cycle 2 day 1. H-scores were calculated as the sum of the products of the percentage of positive staining areas and the staining intensity (0, 1, 2 or 3), and ranged from 0 to 300. A score of 0 represents the absence of expression, and an H-score of 300 represents maximum expression.

H- Score Pre-Treatment
GroupValue95% CI
Study Participants236.47± 67.25
H-Score Post-Treatment
GroupValue95% CI
Study Participants238.32± 39.56
Change in Ki-67 With Treatment Secondary · Baseline (Pre-Treatment) and Cycle 2 Day 1, each Cycle is 28 days

Ki-67 was calculated as a percentage cells staining positive by Immunohistochemistry (IHC).

Pre-Treatment
GroupValue95% CI
Study Participants13.61± 10.18
Post-Treatment
GroupValue95% CI
Study Participants19.11± 17.83

Adverse events — posted to ClinicalTrials.gov

Time frame: 3 years and 5 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Study Participants
Serious: 7/20 (35%)
Deaths: 11/20

Serious adverse events (9 terms)

ReactionSystemStudy Participants
Upper Gastrointestinal HemorrhageGastrointestinal disorders
Acute Kidney InjuryRenal and urinary disorders
Febrile NeutropeniaBlood and lymphatic system disorders
HypoglycemiaMetabolism and nutrition disorders
Thromboembolic EventVascular disorders
HypokalemiaMetabolism and nutrition disorders
PneumothroaxRespiratory, thoracic and mediastinal disorders
Non-cardiac Chest PainGeneral disorders
EncephalopathyNervous system disorders
Other adverse events (47 terms — click to expand)

ReactionSystemStudy Participants
Neutrophil Count IncreasedInvestigations
NauseaMetabolism and nutrition disorders
AnemiaBlood and lymphatic system disorders
FatigueGeneral disorders
Platelet Count DecreasedInvestigations
Creatinine IncreasedInvestigations
VomitingGastrointestinal disorders
Alanine Aminotransferase IncreasedInvestigations
AnorexiaMetabolism and nutrition disorders
DiarrheaGastrointestinal disorders
Aspartate Aminotransferase IncreasedInvestigations
Weight LossInvestigations
DyspneaRespiratory, thoracic and mediastinal disorders
Rash Maculo-papularSkin and subcutaneous tissue disorders
Edema LimbsGeneral disorders
DysguesiaNervous system disorders
FeverGeneral disorders
Alkaline Phosphatase IncreasedInvestigations
HypophosphatemiaMetabolism and nutrition disorders
Blood Bilirubin IncreasedInvestigations
HeadacheNervous system disorders
Peripheral Sensory NeuropathyNervous system disorders
HypomagnesemiaMetabolism and nutrition disorders
PruritusSkin and subcutaneous tissue disorders
ConstipationGastrointestinal disorders
Urinary Tract InfectionInfections and infestations
Investigations: Other- HyperphosphatemiaInvestigations
HyperphosphatemiaMetabolism and nutrition disorders
Acute Kidney InjuryRenal and urinary disorders
Upper Respiratory InfectionInfections and infestations
Nasal CongestionRespiratory, thoracic and mediastinal disorders
Skin infectionInfections and infestations
MalaiseGeneral disorders
Back PainMusculoskeletal and connective tissue disorders
GastroparesisGastrointestinal disorders
Edema FaceGeneral disorders
HematomaVascular disorders
Abdominal DistentionGastrointestinal disorders
HyperglycemiaMetabolism and nutrition disorders
Dry SkinSkin and subcutaneous tissue disorders

Most-reported serious reactions: Upper Gastrointestinal Hemorrhage, Acute Kidney Injury, Febrile Neutropenia, Hypoglycemia, Thromboembolic Event, Hypokalemia, Pneumothroax, Non-cardiac Chest Pain.

Data from ClinicalTrials.gov NCT02420691 adverse events section.

Sponsor's own description

This phase II trial studies how well ribociclib works in treating patients with neuroendocrine tumors of the foregut, which includes the thymus, lung, stomach, and pancreas, that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced tumors). Ribociclib 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. Cyclin D1, cancer progression, and opportunities in cancer treatment.
    Qie S, Diehl JA. · · 2016 · cited 527× · PMID 27695879 · DOI 10.1007/s00109-016-1475-3
  2. Intrinsic and acquired resistance to CDK4/6 inhibitors and potential overcoming strategies.
    Xu XQ, Pan XH, Wang TT, Wang J, et al · · 2021 · cited 54× · PMID 32504067 · DOI 10.1038/s41401-020-0416-4
  3. Gene Expression Signatures Identify Novel Therapeutics for Metastatic Pancreatic Neuroendocrine Tumors.
    Scott AT, Weitz M, Breheny PJ, Ear PH, et al · · 2020 · cited 47× · PMID 31937620 · DOI 10.1158/1078-0432.ccr-19-2884
  4. Targeted therapy of gastroenteropancreatic neuroendocrine tumours: preclinical strategies and future targets.
    Aristizabal Prada ET, Auernhammer CJ. · · 2018 · cited 24× · PMID 29146887 · DOI 10.1530/ec-17-0286
  5. Systemic treatment for lung carcinoids: from bench to bedside.
    Torniai M, Scortichini L, Tronconi F, Rubini C, et al · · 2019 · cited 19× · PMID 31273555 · DOI 10.1186/s40169-019-0238-5
  6. Biological Hallmarks and New Therapeutic Approaches for the Treatment of PDAC.
    Digiacomo G, Volta F, Garajova I, Balsano R, et al · · 2021 · cited 16× · PMID 34440587 · DOI 10.3390/life11080843
  7. Recent advances of cyclin-dependent kinases as potential therapeutic targets in HR+/HER2- metastatic breast cancer: a focus on ribociclib.
    Edessa D, Sisay M. · · 2017 · cited 11× · PMID 29263697 · DOI 10.2147/bctt.s150540
  8. Biologics in gastrointestinal and pancreatic neuroendocrine tumors.
    Liu IH, Kunz PL. · · 2017 · cited 9× · PMID 28736633 · DOI 10.21037/jgo.2016.12.09

Verify or expand the search:

Other trials of Laboratory Biomarker Analysis

Trials testing the same drug.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02420691.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing