E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Familial adenomatous polyposis |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059327 |
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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 assess the efficacy of a new oral formulation of 2-PPA (PEAC minitablet), given as a 6-months treatment of colorectal polyps in patients with familial adenomatous polyposis (FAP) in comparison to a placebo treatment. |
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E.2.2 | Secondary objectives of the trial |
To assess the safety of a new oral formulation of 2-PPA (PEAC minitablet), given as a 6-months treatment of colorectal polyps in patients with familial adenomatous polyposis (FAP) in comparison to a placebo treatment. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- age equal or higher than 16 years - diagnosis of FAP confirmed either by molecular analysis of the APC gene (presence of an APC mutation) or by characteristic family history or by the presence of more than 100 colorectal adenomatous polyps before colectomy - presence of more than 5 colorectal polyps of at least 2 mm in diameter after colectomy - they are not yet colectomized or previous colectomy was performed more than 2 years ago - preventive surgery is not planned for the forthcoming 8 months - their performance status suffices grade 2 of the WHO criteria - females must have a negative pregnancy test - absence of clinically relevant hematopoietic, hepatic, pancreatic or renal disease - informed consent signed by the patient and by the legally acceptable representative, if required
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E.4 | Principal exclusion criteria |
- history or presence of hepatic disease or any active liver dysfunction (AST > 100 U/L) - presence of a thrombocytopenia (platelets < 100 x 109/L) - history or presence of any bleeding or coagulative disorders - history or presence of any pancreatic disorders - renal insufficiency - metabolic disorders like enzymopathies - acute or chronic bacterial, viral or fungal infection - severe uncontrolled concomitant diseases - known hypersensitivity to 2-propyl pentanoic acid (valproic acid) - concomitant treatment with any other anti-epileptic drug, with mefloquin, cimetidin, erythromycin, fluoxetin, felbamat, codein, barbiturates, primidon, neuroleptic or antidepressive drugs, benzodiazepine, with any anti-coagulative drug or with non-steroidal anti-inflammatory drugs (NSAID’s) such as acetyl salicylic acid. - any treatment of FAP within 2 months prior to inclusion into the study - participation in a clinical trial not related to FAP within 30 days prior to inclusion into the study - pregnant or nursing females (positive pregnancy test immediately prior to inclusion into the study) - females of childbearing potential without using a safe contraceptive measure (e.g. IUD or oral contraceptives; barrier method or condom if used in combination with a spermicide) - alcohol or drug abuse
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint for assessment of efficacy will be • the percentage change in the total number of polyps with a diameter > 2 mm at the end of the 6-months treatment period compared to baseline as determined from video tapes of colonoscopy before and after treatment by three independent physicians qualified in colonoscopy.
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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 | Yes |
E.6.7 | Pharmacodynamic | No |
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 | 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 | 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.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |