Clinical trial NCT01945762: Observational Study to Evaluate Vandetanib in RET -/+ Patients With Metastatic Medullary Thyroid Cancer
Official title: European, Observational, Prospective Study to Evaluate the Benefit/Risk of Vandetanib in RET Mutation Negative and Positive Patients With Symptomatic, Aggressive, Sporadic, Unresectable, Locally Advanced/Metastatic Medullary Thyroid Cancer
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Symptomatic, Aggressive, Sporadic, Unresectable, Locally
Advanced/Metastatic Medullary Thyroid Cancer (MTC)
Keywords: RET Mutation
DIagnostics
Medullary Thyroid Cancer
MTC
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Worldwide Clinical Trials
First posted: 2013-09-19
Enrollment: 31
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Symptomatic, Aggressive, Sporadic, Unresectable, Locally Advanced/Metastatic Medullary Thyroid Cancer (MTC)

Brief summary: This is a European multinational, multicenter, non-interventional (observational) and prospective study. It is carried on to confirm in real life conditions the benefit/risk of vandetanib (CAPRELSA™) 300 mg, both in RET negative and RET positive patients with symptomatic, aggressive, sporadic, unresectable, locally advanced/metastatic MTC.

Detailed description: This is a multinational, multicenter, non-interventional (observational) and prospective study. European countries where vandetanib is on the market will participate in the study.

This study is being conducted to fulfil the specific obligation post-authorisation measure for the conditional marketing authorisation. It is carried on to confirm in real life conditions the benefit/risk of vandetanib (CAPRELSA™) 300 mg, both in RET negative and RET positive patients with symptomatic, aggressive, sporadic, unresectable, locally advanced/metastatic MTC. The clinical benefit of vandetanib (CAPRELSA™) 300 mg has previously been established in a clinical trial (Study 58) on the basis of a clinically and statistically significant advantage in progression free survival (PFS) which was supported by a high response rate and substantial duration of response.

Eligibility criteria: Inclusion Criteria:

1. Signed informed consent 2. Male or female aged 18 years or above 3. Histological diagnosis of MTC 4. Patients with symptomatic and aggressive sporadic MTC, who have unresectable, locally advanced/metastatic disease. (The factors considered by the investigator to determine a patient's disease to be symptomatic and aggressive will be recorded in the CRF). 5. Measurable disease:

* assessment confirmed within the 12 weeks previous to start of treatment, and
* defined according to RECIST 1.1: at least one lesion, not irradiated, that can be accurately measured as ≥10 mm in the longest diameter (except lymph nodes which must have short axis ≥15 mm) with CT or MRI and which is suitable for accurate repeated measurements. Measurable lesions with calcifications should not be assessed as target lesions unless no other measurable lesion is available. 6. Known definite RET mutation status (definition according to section 3.2). The status should be:
* for patients prescribed with vandetanib: positive or negative
* for patients not prescribed with vandetanib: negative RET mutation status must be determined from a tumour sample obtained within 18 months prior to enrollment. It is strongly recommended that a tissue sample obtained within 6 months prior to enrolment is used. 7. For patients newly prescribed vandetanib 300 mg, the prescription should be issued according to marketing authorisation and following the vandetanib Summary of Product Characteristics (SmPC) (Appendix B). The starting dose could be reduced to 200 mg in patients with moderate renal impairment

  * Exclusion criteria

    1. Current or planned inclusion/participation in a clinical trial
    2. Patients already receiving vandetanib or who have received vandetanib for their MTC before the study first visit
    3. Contraindications according to the vandetanib SmPC (not applicable for patients who do not receive vandetanib): (a) Patients with a QT interval corrected for heart rate (QTc) interval over 480 msec: (i) Congenital long QT syndrome (ii) Concomitant use of vandetanib with the following medicinal products known to also prolong the QT interval and / or induce Torsades de pointes: Arsenic, cisapride, erythromycin intravenous (IV), toremifene, mizolastine, moxifloxacin, Class I A and III antiarrhythmics (b) Currently pregnant or breast feeding (c) Hypersensitivity to the active substance or to any of the excipients (d) Severe renal impairment: creatinine clearance \< 30 ml/minute calculated by Cockcroft-Gault formula. (See Appendix D). (e) Serum bilirubin greater than 1.5 x the upper limit of reference range (ULRR) (f) Potassium, magnesium or calcium outside the normal laboratory range

Primary outcomes:
measure: Assessment of Objective Response Rate
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Assessment of Disease control rate
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Assessment of Duration of Response
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Assessment of Progression Free Survival
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Evaluation of Safety by assessment of QTc prolongations
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Evaluation of Safety by assessment of Adverse Events
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Evaluation of Safety by assessment of vital signs
timeFrame: From enrollment until study completion, assessed up to 38 months
measure: Evaluation of Safety by assessment of laboratory data
timeFrame: From enrollment until study completion, assessed up to 38 months
Secondary outcomes:
measure: Patient Characteristics
timeFrame: From enrollment until study completion, assessed up to 38 months
Other outcomes:
Locations:
city: Belgium
country: Belgium
facility: investigational Site Belgium
city: France
country: France
facility: investigational Site France
city: Germany
country: Germany
facility: investigational Site Germany
city: Italy
country: Italy
facility: investigational Site Italy
city: Luxembourg
country: Luxembourg
facility: investigational Site Luxembourg
city: Netherlands
country: Netherlands
facility: investigational Site Netherlands
city: Spain
country: Spain
facility: investigational Site Spain
city: United Kingdom
country: United Kingdom
facility: investigational Site United Kingdom
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: