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NCT00843531

RAD001 and Erlotinib in Patients With Neuroendocrine Tumors

Terminated Phase 2 Results posted Last updated 29 September 2020
What this trial tests

Phase 2 trial testing RAD001 in Neuroendocrine Tumors in 17 participants. Terminated before completion.

Timeline
25 June 2009
Primary endpoint
16 July 2013
20 August 2016

Quick facts

Lead sponsorUniversity of California, San Francisco
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment17
Start date25 June 2009
Primary completion16 July 2013
Estimated completion20 August 2016
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

University of California, San Francisco

Who can join

18 and older, any sex, with Neuroendocrine Tumors. 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.

Objective Response Rate (ORR) Primary · Up to 2 years

Objective response is defined as complete response (CR) or partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. The primary analysis population is based on efficacy-evaluable patients, defined as those patients who receive at least one cycle of RAD001 plus erlotinib and undergo at least one post-baseline response assessment or die while on therapy.

GroupValue95% CI
RAD001 Plus Erlotinib0
Number of Patients With Dose-limiting Toxicity (DLT) Secondary · Up to 9 months

Primary safety analysis will be conducted after the first 16 patients have had potential for completion of two cycles of protocol therapy. All patients receiving at least one dose of protocol therapy will be included in the analyses. All patients with treatment-related, Grade 3 or higher adverse events according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 3 resulting in a DLT will be reported.

GroupValue95% CI
RAD001 and Erlotinib8
Duration of Objective Response Secondary · Up to 2 years

Duration of objective response will be defined as the time from the initial documentation of response to documented disease progression or death from any cause on study

GroupValue95% CI
RAD001 and ErlotinibNA

Adverse events — posted to ClinicalTrials.gov

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

RAD001 Plus Erlotinib
Serious: 4/17 (24%)
Deaths: 6/17

Serious adverse events (12 terms)

ReactionSystemRAD001 Plus Erlotinib
DiarrheaGastrointestinal disorders
Edema: limbVascular disorders
CreatinineInvestigations
Pain - BackMusculoskeletal and connective tissue disorders
Pain - Extremity-limbMusculoskeletal and connective tissue disorders
Incontinence, urinaryRenal and urinary disorders
Renal failureRenal and urinary disorders
Alkaline phosphataseInvestigations
Pain - Other - Right upper quadrant painInvestigations
Pain - Abdomen NOSGastrointestinal disorders
FeverGeneral disorders
Distension/bloating, abdominalGastrointestinal disorders
Other adverse events (48 terms — click to expand)

ReactionSystemRAD001 Plus Erlotinib
DiarrheaGastrointestinal disorders
StomatitisGastrointestinal disorders
FatigueGeneral disorders
Rash: acne/acneiformSkin and subcutaneous tissue disorders
AnorexiaMetabolism and nutrition disorders
Dry SkinSkin and subcutaneous tissue disorders
Weight LossInvestigations
Hemorrhage, pulmonary/upper respiratory - NoseRespiratory, thoracic and mediastinal disorders
DysgeusiaNervous system disorders
PruritusSkin and subcutaneous tissue disorders
HypophosphatemiaMetabolism and nutrition disorders
CreatinineRenal and urinary disorders
Nail changesSkin and subcutaneous tissue disorders
DehydrationMetabolism and nutrition disorders
HypercholesteremiaInvestigations
Dry mouthGastrointestinal disorders
Rash/desquamationSkin and subcutaneous tissue disorders
HyponatremiaMetabolism and nutrition disorders
FlatulenceGastrointestinal disorders
AlopeciaSkin and subcutaneous tissue disorders
LymphopeniaBlood and lymphatic system disorders
AST, SGOT(serum glutamic oxaloacetic transaminase)Investigations
Edema: limbVascular disorders
VomitingGastrointestinal disorders
NauseaGastrointestinal disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)General disorders
Pain - Head/headacheNervous system disorders
Alkaline phosphataseInvestigations
HypomagnesemiaMetabolism and nutrition disorders
HyperglycemiaMetabolism and nutrition disorders
Pain - Abdomen NOSGastrointestinal disorders
Pain - BackMusculoskeletal and connective tissue disorders
HypocalcemiaMetabolism and nutrition disorders
ConstipationGastrointestinal disorders
DysphagiaGastrointestinal disorders
DyspneaCardiac disorders
HemoglobinInvestigations
ChillsGeneral disorders
Neutrophils/granulocytesBlood and lymphatic system disorders
LymphopeniaBlood and lymphatic system disorders

Most-reported serious reactions: Diarrhea, Edema: limb, Creatinine, Pain - Back, Pain - Extremity-limb, Incontinence, urinary, Renal failure, Alkaline phosphatase.

Data from ClinicalTrials.gov NCT00843531 adverse events section.

Sponsor's own description

The purpose of this study is to test how safe and effective the combination of RAD001 and erlotinib is in patients with neuroendocrine tumors.

Publications & conference data

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

  1. Endocrine tumours in neurofibromatosis type 1, tuberous sclerosis and related syndromes.
    Lodish MB, Stratakis CA. · · 2010 · cited 54× · PMID 20833335 · DOI 10.1016/j.beem.2010.02.002
  2. Molecular and therapeutic advances in the diagnosis and management of malignant pheochromocytomas and paragangliomas.
    Lowery AJ, Walsh S, McDermott EW, Prichard RS. · · 2013 · cited 34× · PMID 23576482 · DOI 10.1634/theoncologist.2012-0410
  3. Systemic Treatment Selection for Patients with Advanced Pancreatic Neuroendocrine Tumours (PanNETs).
    Megdanova-Chipeva VG, Lamarca A, Backen A, McNamara MG, et al · · 2020 · cited 19× · PMID 32708210 · DOI 10.3390/cancers12071988
  4. 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
  5. Emerging use of everolimus in the treatment of neuroendocrine tumors.
    Gajate P, Martínez-Sáez O, Alonso-Gordoa T, Grande E. · · 2017 · cited 14× · PMID 28684922 · DOI 10.2147/cmar.s113382
  6. The pivotal role of mammalian target of rapamycin inhibition in the treatment of patients with neuroendocrine tumors.
    Phan AT, Dave B. · · 2016 · cited 11× · PMID 27539383 · DOI 10.1002/cam4.742
  7. Mechanisms of Resistance in Gastroenteropancreatic Neuroendocrine Tumors.
    Shi C, Morse MA. · · 2022 · cited 9× · PMID 36551599 · DOI 10.3390/cancers14246114
  8. An Overview on the Sequential Treatment of Pancreatic Neuroendocrine Tumors (pNETs).
    Alonso-Gordoa T, Díez JJ, Molina J, Reguera P, et al · · 2015 · cited 4× · PMID 27182476 · DOI 10.1007/s40487-015-0007-6

Verify or expand the search:

Other trials of RAD001

Trials testing the same drug.

Other recruiting trials for Neuroendocrine Tumors

Currently open trials in the same condition.

Other University of California, San Francisco trials

Trials by the same sponsor.

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