E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic fatigue syndrome |
Chronisch vermoeidheidssyndroom (CVS) |
|
E.1.1.1 | Medical condition in easily understood language |
Chronic fatigue syndrome |
Chronisch vermoeidheidssyndroom (CVS) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066564 |
E.1.2 | Term | Chronic fatigue |
E.1.2 | System Organ Class | 100000004867 |
|
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 |
To evaluate in CFS patients the effect of sodium oxybate on fatigue |
|
E.2.2 | Secondary objectives of the trial |
To evaluate in CFS patients
- the effect of sodium oxybate on subjective and objective measures of sleep characteristics, sleepiness, general health and pain
- the interdependence of sleep quality and fatigue
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients suffering from chronic, disabling, unexplained fatigue with at least four minor criteria for CFS, according to the 1994 Fukuda case definition
- Negative screening for FMS according to the American College of Rheumatology (ACR) classification criteria
- 18 ≤ age ≤ 65
- Female patient of childbearing potential (premenopausal female biologically capable of becoming pregnant) has a confirmed negative pregnancy test at the start of the trial and has to employ a double barrier method of contraception
- Written, signed and dated informed consent must be obtained from each patient
- Patient able to understand and to comply with the requirements of the study
-Willing to abstain from taking any medication or treatment prohibited in the protocol
-Willing to abstain from using alcohol |
|
E.4 | Principal exclusion criteria |
- FMS
- Presence of an inflammatory rheumatic disease or a painful disorder other than FMS
- Fatigue that is explained by established medical or psychiatric causes
- 18 > age > 65
- Apnea/hypopnea index (AHI) ≥ 15 on PSG (exempted if receiving continuous positive airway pressure CPAP with documented good compliance and control of AHI on PSG)
- Any other respiratory disease (chronic obstructive pulmonary disease, asthma,…)
- Deficiency in succinic semialdehyde dehydrogenase
- Porphyria
- Unsufficiently controlled hypertension (≥ 140/90 mmHg)
- Heart failure
- Liver or kidney disease (hepatitic transaminases AST and ALT > 5x upper limit of normal ULN (graad 3 AE), Total bilirubin > 1.5 mg/dl (graad 3 AE) of normal direct bilirubin; serum creatinine level > 2.0 mg/dl)
- Any primary neurological disease, seizure disorder or epilepsy
- Any past history of narcolepsy
- Sodium oxybate treatment in the past
- Any history of substance abuse (including abuse of alcohol)
- Inability to withdraw from psychoactive drugs
- Current use of sedatives (benzodiazepine and non-benzodiazepine GABA-ergic agonists), opioids or antidepressant medication or a drug-free interval of less than 4 weeks
- Female patient who is pregnant, lactating or has a positive pregnancy test result
- Inability to adopt a double barrier method of contraception
- Occupation that requires night-shift work |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Effect of sodium oxybate on fatigue |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Polysomnography (PSG): on night 42 and 112
Multiple Sleep Latency Test (MSLT): on day 43 and 113 |
|
E.5.2 | Secondary end point(s) |
- Effect of sodium oxybate on subjective and objective measures of sleep characteristics, sleepiness, general health and pain
- the interdependence of sleep quality and fatigue
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Questionnaires: on day 43 and 113 |
|
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 | 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) | 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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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
|
Last visit of the last subject |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |