Propionic acidaemia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:35 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Propionic acidaemia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study to evaluate the long-term safety and effectiveness of mRNA-3927 in patients with Propionic Acidemia https://clinicaltrials.eu/trial/a-study-to-evaluate-the-long-term-safety-and-effectiveness-of-mrna-3927-in-patients-with-propionic-acidemia/ Wed, 29 Apr 2026 15:08:58 +0000 https://clinicaltrials.eu/trial/a-study-to-evaluate-the-long-term-safety-and-effectiveness-of-mrna-3927-in-patients-with-propionic-acidemia/ This study focuses on Propionic Acidemia, which is a rare genetic disorder that prevents the body from properly breaking down certain proteins. This condition can lead to a buildup of acid in the blood, which may cause health problems. The study aims to evaluate the long-term safety and clinical activity of mRNA-3927, an experimental treatment made of modified messenger ribonucleic acid. This substance is delivered through intravenous injection, which means it is administered directly into a vein, and it is packaged in lipid nanoparticles, which are tiny fat-based particles used to protect the medicine as it enters the body.

Participants in this study have previously been part of an earlier clinical trial. During this phase, they will continue to receive the treatment to monitor how the body reacts over a longer period. The focus is on tracking any side effects or changes in health to ensure the treatment remains safe for long-term use.

]]> Study on the Safety and Effectiveness of mRNA-3927 for Patients with Propionic Acidemia https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-mrna-3927-for-patients-with-propionic-acidemia/ Wed, 29 Apr 2026 14:28:50 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-mrna-3927-for-patients-with-propionic-acidemia/ This clinical trial is focused on studying a rare genetic condition called Propionic Acidemia. This condition affects the body’s ability to break down certain proteins and fats, leading to a buildup of harmful substances. The study will use a new treatment called mRNA-3927, which is a type of gene therapy. This treatment involves using modified messenger ribonucleic acid (mRNA) to help the body produce specific proteins that are missing or not working properly in people with Propionic Acidemia. The mRNA is delivered into the body using tiny particles called lipid nanoparticles.

The purpose of the study is to evaluate the safety and effectiveness of mRNA-3927 in people with Propionic Acidemia. Participants will receive the treatment through an intravenous (IV) infusion, which means it will be administered directly into a vein. The study will be conducted in two parts. In the first part, the focus will be on assessing the safety and tolerability of the treatment. In the second part, the study will look at how well the treatment works by comparing the frequency of metabolic decompensation events (MDEs) in participants receiving the treatment to those receiving standard care.

Throughout the study, participants will be monitored for any side effects and changes in their condition. The study will also measure various biological markers in the blood to understand how the treatment affects the body. This trial aims to provide valuable information about the potential benefits and risks of using mRNA-3927 as a treatment for Propionic Acidemia, which could lead to new therapeutic options for individuals affected by this condition.

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