Familial adenomatous polyposis MedDRA version: 7.1 Level: LLT Classification code 10059327
Conditions
Interventions
Product Name: PEAC
Pharmaceutical Form: Granules
INN or Proposed INN: Valproic Acid, Sodium
Current Sponsor code: VPA
Other descriptive name: 2-PPA, 2-pentanoic acid
Concentration unit: mg milligram(s
Sponsors
TopoTarget Germany AG
Eligibility
Sex/Gender
All
Inclusion criteria
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 Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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.;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. | — |
Countries
Denmark
Outcome results
None listed