Glycerol Phenylbutyrate for Pyruvate Dehydrogenase Deficiency

We are studying whether Glycerol Phenylbutyrate can help reduce fatigue and improve quality of life for patients with Pyruvate Dehydrogenase Deficiency. The trial will also assess its safety and effects on neurodevelopmental symptoms.

1-2 years≤5 visitsEfficacy phase (II)No PlaceboStandard MedicinesOralEndocrinologyGastroenterologyNeurology

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Ravicti
Ravicti is a medicine used to help lower high ammonia levels in people with urea cycle disorders.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Glycerol Phenylbutyrate
Glycerol phenylbutyrate is a substance that helps lower high ammonia levels in people with urea cycle disorders.

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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Centre Hospitalier Universitaire De Lille
Pédiatrie
Loos, France
Centre Hospitalier Universitaire De Montpellier
Neurométabolisme
Montpellier, France
Assistance Publique Hopitaux De Paris
Maladies Métaboliques
Montrouge, France
Sponsor: Assistance Publique Hopitaux De Paris
Last updated: Nov 27, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.