Pyruvate dehydrogenase complex deficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Thu, 28 May 2026 11:27:23 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Pyruvate dehydrogenase complex deficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of glycerol phenylbutyrate (oral liquid) for treatment of fatigue in patients with pyruvate dehydrogenase deficiency https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-glycerol-phenylbutyrate-for-patients-with-pyruvate-dehydrogenase-deficiency/ Wed, 29 Apr 2026 15:04:28 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-glycerol-phenylbutyrate-for-patients-with-pyruvate-dehydrogenase-deficiency/ This clinical trial focuses on studying Pyruvate Dehydrogenase Deficiency, a rare genetic condition that affects how the body processes certain nutrients. The study will test a medication called Glycerol Phenylbutyrate (also known as RAVICTI), which is given as a liquid that can be taken by mouth or through feeding tubes.

The purpose of this research is to evaluate how well Glycerol Phenylbutyrate works in reducing tiredness and fatigue in people who have Pyruvate Dehydrogenase Deficiency. The study will last for 6 months, during which participants will take the medication daily. The maximum daily dose of the medication will be 12 grams.

The treatment will be given as a liquid medicine that can be taken orally or administered through a nasogastric tube or gastrostomy tube (a feeding tube that goes directly into the stomach). Throughout the study, doctors will monitor how the treatment affects participants’ energy levels and overall well-being using specialized questionnaires that measure fatigue levels.

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