E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cataplexy in adult patients with narcolepsy |
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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 monitor for endocrine changes in response to treatment of cataplexy with Xyrem · To focus on the hypothalamic pituitary axis
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E.2.2 | Secondary objectives of the trial |
· To confirm the safety of Xyrem on potential endocrine changes |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
To be eligible to participate in this study, all of the following criteria must be met:
· Male or female subjects, aged from 18 to 65 years inclusive, from any racial group; · Written informed consent dated and signed by the subject; · The subject must have met criteria for a primary diagnosis of narcolepsy with cataplexy: · Daytime sleepiness daily for at least 3 months · Inadvertent naps / Sleep attacks · Bilateral postural muscle weakness triggered by emotions; · The subject must demonstrate an adequate need for cataplexy treatment, as determined by the Investigator, based on a review of the subject’s history and any relevant clinical findings. The subject must either have a need for, or have had an adequate response to standard treatment as determined by the treating physician; · Female subjects without childbearing potential (2 years post-menopausal, bilateral oophorectomy or tubal ligation, complete hysterectomy) are eligible. Female subjects with childbearing potential are eligible if they are sexually abstinent or if they use a medically accepted contraceptive method (hormonal contraception must be stable since at least 3 months before entry in the study and must be continued until the end of the study because of the influence on IGF-1 levels); · Be considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.
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E.4 | Principal exclusion criteria |
Subjects must be excluded if they meet any of the following criteria:
· History or presence of cardiovascular, respiratory, hepatic/biliary, renal, gastrointestinal, endocrinological, neurological disorders capable of altering the absorption, distribution, metabolism or elimination of drugs, or constituting a possible risk factor when taking the study medication. A blood sampling and urinalysis taken within the 3 months preceding the screening or at screening must demonstrate there is no risk according to the investigator’s judgment to include the subject (see section 11.1.7); · History of clinically significant hypersensitivity or allergy to sodium oxybate or to any of the excipients; · History or presence of drug addiction (positive drug screen), or excessive use of alcohol (positive alcohol breath test); · Patients with succinic semialdehyde dehydrogenase deficiency; · Patients being treated with sedative hypnotics, other CNS depressants, corticoids (which are not taken at a stable dose for at least two months. No topical, intra-muscular or intra-venous formulation), opioids or barbiturates during one week (one month for enzyme-inducers) preceding the study drug administration; · Participation in another clinical trial less than 3 months before the present study; · Subjects who, in the judgment of the investigator, are likely to be non reliable or non-compliant or uncooperative during the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
· IGF-1: Single daily measurement at Baseline, month 1 and month 3 visits, in fasting conditions, around 6 am (ca 8 h after bedtime dose) |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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The end of study is defined as the date of database lock. |
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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 | |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |