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Gemcitabine - Oxaliplatin for Advanced Refractory Thyroid Cancer Patients: a Phase II Study

Phase 2
Terminated
Conditions
Thyroid Cancer
Interventions
Registration Number
NCT02472080
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Brief Summary

Radioiodine refractory differentiated thyroid cancer is a rare tumor and therapeutic options are limited in this setting. Molecular targeted therapies have recently been developed for progressive disease and demonstrated clinical activity, especially with anti-angiogenic agents.

For patients with contra-indication to these agents or in case of progression or toxicity during treatment, chemotherapy is usually proposed but this strategy has not been validated by prospective data.

The investigators propose to conduct an open single arm phase 2 study to evaluate response rate according to RECIST 1.1 with GEMOX regimen (gemcitabine - oxaliplatin combination) for advanced radioiodine refractory differentiated thyroid cancer patients after anti-angiogenic agents or in case of contra-indication to anti-angiogenic therapy.

Detailed Description

The aim is to study efficacy of chemotherapy with gemcitabine - oxaliplatin combination for advanced radioiodine refractory differentiated thyroid cancer patients after anti-angiogenic agents or in case of contra-indication to anti-angiogenic therapy.

For refractory thyroid cancer patients, in case of contra-indication to molecular targeted therapies or in case of progression or toxicity during treatment, alternative treatments are urgently needed.

Our study could precise if cytotoxic agents should remain a treatment option for refractory thyroid cancer after molecular targeted therapies or in case of contra-indication.

Numerous clinical trials are proposed to evaluate various molecular targeted therapies for refractory thyroid cancer, either conducted by institutional or industrial promoters. In contrast, chemotherapy trials are lacking. As generics are actually available for gemcitabine and oxaliplatin, no industrial support can be expected and prospective evaluation a chemotherapy regimen need to be conducted by an institutional promoter ("Assistance Publique - Hôpitaux de Paris"), supported by many French expert teams through the rare tumor network ""TUTHYREF"".

GEMOX regimen will be administrated intravenously every two weeks in ambulatory setting.

The objective of this study is to analyse the efficacy of chemotherapy with gemcitabine - oxaliplatin combination for advanced radioiodine refractory differentiated thyroid cancer patients after anti-angiogenic agents or in case of contra-indication to anti-angiogenic therapy.

The primary end point is overall response rate (complete + partial response - CR+PR), according to RECIST 1.1, assessed by local investigator at 4 months.

30 patients will be included in a two steps design (Fleming design). A response rate lower than 15% define the inefficacy level. Minimal efficacy should be 35% response rate.

First step: inclusion of 15 patients. If 2 or less responses are observed: end of study for inefficacy If 6 or more responses are observed: efficacy endpoint has been reached If 3 to 5 responses are observed, additional inclusions are needed. Second step: inclusion of 15 additional patients. Nine responses are expected to define a positive study. Details are presented in statistical section.

The secondary end points are:

* Safety report according to NCI CTCAE v 4.0 grading scale

* Early metabolic response rate on 18F-FDG-PET/CT at 2 months

* Disease control rate (DCR = CR + PR + SD) ≥ 6 months

* Duration of response

* Time to progression

* Progression free survival

* Overall survival.

* Evolution of quality of life according to Fact-G and EQ-5D "

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
21
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
gemcitabine -oxaliplatine combinationgemcitabine -oxaliplatine combination-
Primary Outcome Measures
NameTimeMethod
overall response rate4 months

overall response rate measured (complete and partial responses) by CT-scan according to RECIST criteria v1.1

Secondary Outcome Measures
NameTimeMethod
number of adverse events and serious adverse events (AE) according to CTCAE v4.03up to 12 month

recording number of adverse events and serious adverse events (AE) according to CTCAE v4.03, during chemotherapy and follow up.

Early metabolic response rate on 18F-FDG-TEP/CT2 months

semi-quantitative PET Response Criteria in Solid Tumors PERCIST1.0 "modified" using SUVmax and body-weight. Central reviewing of FDG-PET data by two readers at the end of the study will be done.

Disease control rate4 months

Disease control rate (DCR) is defined as the percentage of patients who have achieved complete response (CR), partial response (PR) or stable disease (SD).

Time to progressionup to 36 months

Time from documentation of tumor response to disease progression or date of death from any cause

Progression free survivalup to 36 months

Time from documentation of tumor response to disease progression or date of death from any cause

Overall survivalup to 36 months

Delay between inclusion and death from any cause

Quality of lifeat day 1(week 1, i.e first cycle) and repeated every 28 days (i.e at W5, W9, W13, W17) during chemotherapy and every 3 months until 12 months

evolution of quality of life will be assessed by the scores at the FACT-G and EQ-5D surveys (French version)

Trial Locations

Locations (1)

Groupe Hospitalier Pitié Salpetriere

🇫🇷

Paris, France

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