E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Investigative Techniques [E05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036706 |
E.1.2 | Term | Primary liver cancer non-resectable |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
This project seeks to determine the potential of hyperpolarized pyruvate MRI in early characterization of primary liver cancer - particularly small tumors. |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• All participants: - Aged 18 – 85 years. The age and gender of the healthy controls of the main study will be matched to the participating patients. - Pre-menopausal women must be confirmed non-pregnant by an onsite test • HCC group: - Diagnosed HCC larger than 10 mm confirmed by standard MRI, or CT or confirmed by tumour biopsy. - Subgroup 1: Not elected for liver surgery - Subgroup 2: Elected for liver surgery
• Small tumor group: - One or more small lesions in the liver requiring 2 years of repeated follow-up as per the EASL guideline. Could be in a liver with a confirmed HCC. |
|
E.4 | Principal exclusion criteria |
• Contraindications for MRI with contrast: - Chronic kidney disease - Patients with tumors must have eGFR > 30 ml/min/1.73m2 - Significant cardiac disease (as left ventricular outflow obstruction or heart failure) - Significant obstructive lung disease or severe asthma - Pacemaker, neurostimulator or cholera implant - Metal foreign bodies such as fragments and irremovable piercings - Unsafe medical implants (safety of heart valves, hips and the like must be confirmed) - Claustrophobia - Largest circumference including arms > 160 cm • Competing systemic disease including diabetes (hypertension, dyslipidemia, diabetes etc. allowed) • Hepatic encephalopathy ≥ grade 2. • Stents or other devices implanted close to the liver that may cause imaging artefacts • Allergy to pyruvate |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Liver metabolism as assessed with 13C label exchange from hyperpolarized pyruvate to bicarbonate, lactate and alanine. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Disease characterization from clinical examination, interviews and patient records. For the group of patients with small tumours, a clinical two year follow-up is required for a final diagnosis. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
After the scan and up to two years after. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
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
|
LVLS plus two years of clinical follow-up for the small tumor patients. |
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |