E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic lateral sclerosis Transient ischemic attack |
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E.1.1.1 | Medical condition in easily understood language |
Amyotrophic lateral sclerosis Mini stroke |
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002026 |
E.1.2 | Term | Amyotrophic lateral sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 detect and characterize metabolic impairment in patients with amyotrophic lateral sclerosis and patients with a transient ischemic attack with a new MRI technique termed Hyperpolarized Pyruvate MRI. |
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E.2.2 | Secondary objectives of the trial |
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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 patients in ALS and TIA groups - Pre-menopausal women must be confirmed non-pregnant by an onsite test
ALS group: - Fulfilling the 2015 revised El Escorial criteria - Upper motor neuron symptoms
TIA group: - TIA within the last 10 days. TIA is defined as symptoms shorter than 24 hours (time definition) with a likely vascular genesis (mimics such as urinary tract infection, hypoglycemia and migraine are excluded) - Symptoms must include unilateral weakness of face and/or limb - Symptom duration > 10 minutes
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E.4 | Principal exclusion criteria |
Contraindications for MRI with contrast: - Chronic kidney disease - Significant cardiac disease such as severe left ventricular outflow obstruction - 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) - Intracranial clips or coils - Claustrophobia - Largest circumference including arms > 160 cm
Competing neurological, psychiatric, liver or systemic disease including diabetes (except prior TIA, unspecific brain MRI findings, hypertension, atherosclerosis and hyperlipidemia/cholesterolemia which are allowed) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Cerebral metabolism as assessed with 13C label exchange from hyperpolarized pyruvate to bicarbonate, lactate and alanine. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Disease characterization from clinical examination, interviews and patient records. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | |