E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Anaplastic Thyroid Carcinoma |
anaplastisch schildkliercarcinoom |
|
E.1.1.1 | Medical condition in easily understood language |
anaplastic thyroid cancer |
anaplastische schildklierkanker |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To investigate the effect of treatment with human recombinant IL1Ra (rhIL1Ra, Anakinra) on health related quality of life (HRQoL), tumor progression and overall survival in patients with anaplastic thyroid carcinoma. |
Onderzoek naar het effect van de behandeling met anakinra in patienten met anaplastisch schildkliercaricnoom. Specifiek wordt het effect op kwaliteit van leven, tumor progressie en overleving. |
|
E.2.2 | Secondary objectives of the trial |
To investigate the effect of treatment with IL1Ra (Anakinra) in patients with ATC on;
- performance status, measured with the Eastern Cooperative Oncology Group (ECOG) performance scale.
- progression free survival (PFS): which is defined as the time interval from date of official diagnosis to date of first progression or death due to any cause, if death occurs before a progression is documented. Progression will be defined according to RECIST criteria.
- safety in terms of occurrence and severity of adverse events according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (NCI CTCAE) (35),
- Measurements of (systemic) inflammation.
|
Het effect van anakinra op performance, progressie-vrije overleving, veiligheid in termen van bijwerkingen, en metingen ten aanzien van systemische ontsteking. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- newly diagnosed patients with a proven diagnosis (cytology or histology) of ATC, for whom surgery is not feasible.
- Patients with proven ATC (cytology or histology), presenting with metastasis not amendable for surgery.
- age ≥ 18 years
|
- ouder dan 18 jaar.
- nieuwe diagnose anaplastisch schildkliercarcinoom, voor wie operatief ingrijpen geen optie meer is.
- iemand met anaplastisch schildkliercarcinoom, die zich presenteert met metastasen en voor wie operatie geen optie meer is. |
|
E.4 | Principal exclusion criteria |
- unable to give informed consent
- pregnancy or breast feeding
- neutropenia (absolute neutrophil count (ANC)< 1.5 x10^9/l)
- patients with a known history of allergic reactions to compounds of similar chemical or biological composition to Anakinra.
- Any severe condition which could interfere with participation in this trial, including, but not limited to: severe renal dysfunction (creatinin clearance < 15 ml/min), severe cardiac failure, severe respiratory conditions etc.
- Active infection; However, after adequate treatment of infections, patients will be eligible for inclusion again.
|
- geen toestemming
- zangerschap of borstvoeding
- neutropenie (ANC<1.55 x10^9/l
- patienten met allergie tegen anakinra of soortgelijk middel
- ernstige aandoening die meedoen aan deze studie kan beïnvloeden, onder andere ernstig nerfalen, hartfalen of ernstige respiratoire aandoeningen.
-actieve infectie (na adequate behandeling kunnen patienten alsnog deelnemen) |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Health Related Quality of Life (HR-QoL): defined as outcome of the QoL life questionnaire specifically designed for anaplastic thyroid cancer patients
Tumor dimensions/tumor progression: TNM classification and tumor size etc. This will be assessed using radiological examinations using the Response Evaluation Criteria in Solid Tumors (RECIST) criteria .
Overall survival (OS): defined as time interval from date of official inclusion to death due to any cause. |
kwaliteit van leven
tumor dimensies/tumor progressie
overleving |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
4, 8 , 12 and 24 weeks after start of treatment imaging studie and laboratory tests to assess tumor status/tumor progression
bi-weekly QoL questionaires . |
4, 8, 12, 24 weken tijdens behandeling vindt beeldvorming en bloedafname plaats om de tumor status/tumor progressie te beoordelen.
Twee-wekelijks vragenlijst naar kwaliteit van elven. |
|
E.5.2 | Secondary end point(s) |
- Improved quality of life (QoL): defined as outcome of the QoL life questionnaire specifically designed for thyroid cancer patients, the THYCA-QoL in combination with the EORTC QLQ-C30.
- The Eastern Cooperative Oncology Group (ECOG) performance status.
- Progression free survival (PFS) based on imaging studies according to RECIST.
- Effect of anakinra on Inflammatory parameters:
• Systemic: CRP, fibrogen, feirritin, triglycerides, albumin, alfa-1-antitripsin, leukocyte counts, IL-6 levels
• Cellular level: quantification and phenotyping of circulating immune cell populations; cytokine production in Peripheral Blood Mononuclear Cells (PBMCs).
|
QoL vragenlijst:
ECOG performance status
RECIST: reductie tumorvolume
inflammatoire parameters |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
4, 8 , 12 and 24 weeks after start of treatment imaging studies to assess tumor status/tumor progression
every month QoL questionaires and blood donations. |
4, 8, 12, 24 weken na behandeling vindt beeldvorming plaats om de tumor status/tumor progressie te beoordelen.
Maandelijks vindt bloedonderzoek en afname van QoL vragenlijsten plaats. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |