Symptomatic, Aggressive, Sporadic, Unresectable, LocallyCaprelsa104NCT01945762
Observational Study to Evaluate Vandetanib in RET -/+ Patients With Metastatic Medullary Thyroid Cancer
An observational study of Symptomatic, Aggressive, Sporadic, Unresectable, Locally and Advanced/Metastatic Medullary Thyroid Cancer (MTC), run by Genzyme, a Sanofi Company at 8 sites in 8 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Symptomatic, Aggressive, Sporadic, Unresectable, Locally, Advanced/Metastatic Medullary Thyroid Cancer (MTC)
- What is being tested?
- Vandetanib 300 mg
- Type of treatment
- Pill or oral medicine
- How many people
- 31 participants
- Where
- 8 sitesin 8 countries
- Study dates
- 17 Feb 2014to 18 Jun 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with Symptomatic, Aggressive, Sporadic, Unresectable, Locally Advanced/Metastatic Medullary Thyroid Cancer (MTC)
Who may be able to join
- 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
Who cannot join
- Current or planned inclusion/participation in a clinical trial
- Patients already receiving vandetanib or who have received vandetanib for their MTC before the study first visit
- 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
Trial locations and contacts
8 sites in 8 countries.
8 sites in 8 countries
United Kingdom1 site
investigational Site United Kingdom
United Kingdom
About this study
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
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.
Read the 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.
- Conditions
- Symptomatic, Aggressive, Sporadic, Unresectable, Locally, Advanced/Metastatic Medullary Thyroid Cancer (MTC)
- Being tested
- Vandetanib 300 mg
- Keywords
- RET Mutation, DIagnostics, Medullary Thyroid Cancer, MTC
- Medical subject headings
- Aggression, Carcinoma, Medullary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vandetanib 300 mgDrug
Also called ZD6474, CAPRELSA
Vandetanib commercial tablets
| Study group | Role | What they receive |
|---|---|---|
| 1. patient cohorts (40 patients/cohort) | RET positive patient cohorts Receives: Drug: Vandetanib 300 mg | |
| 2. patient cohorts (40 patients/cohort) | RET negative patient cohorts Receives: Drug: Vandetanib 300 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of Objective Response Rate
Assessment of Objective Response Rate [using Response Evaluation Criteria In Solid Tumours (RECIST) 1.1]
Measured: From enrollment until study completion, assessed up to 38 months
Assessment of Disease control rate
Assessment of Disease control rate [using Response Evaluation Criteria In Solid Tumours (RECIST) 1.1]
Measured: From enrollment until study completion, assessed up to 38 months
Assessment of Duration of Response
Assessment of Duration of Response (using RECIST 1.1)
Measured: From enrollment until study completion, assessed up to 38 months
Assessment of Progression Free Survival
Assessment of Progression Free Survival (using RECIST 1.1)
Measured: From enrollment until study completion, assessed up to 38 months
Evaluation of Safety by assessment of QTc prolongations
Assessment of QTc prolongations
Measured: From enrollment until study completion, assessed up to 38 months
Evaluation of Safety by assessment of Adverse Events
Assessment of Adverse Events
Measured: From enrollment until study completion, assessed up to 38 months
Evaluation of Safety by assessment of vital signs
Assessment of Vital signs
Measured: From enrollment until study completion, assessed up to 38 months
Evaluation of Safety by assessment of laboratory data
Assessment of Laboratory data
Measured: From enrollment until study completion, assessed up to 38 months
Secondary outcomes (1)
Patient Characteristics
Patient demographics and medical history / Disease characteristics / Death / Treatment information
Measured: From enrollment until study completion, assessed up to 38 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2013 (estimated)
- Study start17 Feb 2014
- Primary completion18 Jun 2020
- Study completion18 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Symptomatic, Aggressive, Sporadic, Unresectable, LocallyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01945762
- Sponsor's protocol ID
- D4200C00104
- Other IDs
- OBS14778
- Acronym
- Caprelsa104
- Status last verified
- Dec 2024
- First submitted
- 10 Sep 2013
- Last updated
- 18 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01945762
- Acronym / sponsor ID
- Caprelsa104 / D4200C00104
- Conditions
- Symptomatic, Aggressive, Sporadic, Unresectable, Locally, Advanced/Metastatic Medullary Thyroid Cancer (MTC)
- Keywords
- RET Mutation, DIagnostics, Medullary Thyroid Cancer, MTC
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Study start
- 17 Feb 2014 (actual)
- Primary completion
- 18 Jun 2020 (actual)
- Study completion
- 18 Jun 2020 (actual)
- First posted
- 19 Sep 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1. patient cohorts (40 patients/cohort) | – | Drug: Vandetanib 300 mgRET positive patient cohorts |
| 2. patient cohorts (40 patients/cohort) | – | Drug: Vandetanib 300 mgRET negative patient cohorts |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Symptomatic, Aggressive, Sporadic, Unresectable, Locally Advanced/Metastatic Medullary Thyroid Cancer (MTC)
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vandetanib 300 mgAlso: ZD6474, CAPRELSAVandetanib commercial tablets | Drug | 1. patient cohorts (40 patients/cohort), 2. patient cohorts (40 patients/cohort) |
Primary outcome
Assessment of Objective Response Rate
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Objective Response Rate [using Response Evaluation Criteria In Solid Tumours (RECIST) 1.1]
Assessment of Disease control rate
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Disease control rate [using Response Evaluation Criteria In Solid Tumours (RECIST) 1.1]
Assessment of Duration of Response
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Duration of Response (using RECIST 1.1)
Assessment of Progression Free Survival
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Progression Free Survival (using RECIST 1.1)
Evaluation of Safety by assessment of QTc prolongations
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of QTc prolongations
Evaluation of Safety by assessment of Adverse Events
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Adverse Events
Evaluation of Safety by assessment of vital signs
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Vital signs
Evaluation of Safety by assessment of laboratory data
Time frame: From enrollment until study completion, assessed up to 38 months
Assessment of Laboratory data
1 secondary outcome
Patient Characteristics
Time frame: From enrollment until study completion, assessed up to 38 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2024.
- OBS14778 clinical study synopsis and addendumLink provided by the sponsor.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
8 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Symptomatic, Aggressive, Sporadic, Unresectable, Locally and Advanced/Metastatic Medullary Thyroid Cancer (MTC), run by Genzyme, a Sanofi Company at 8 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Belgium, France, Germany, Italy, Luxembourg and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of Objective Response Rate (time frame: From enrollment until study completion, assessed up to 38 months). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2024.