Methylmalonic acidaemia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:32:19 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Methylmalonic acidaemia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on mRNA-3705 for Patients with Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency https://clinicaltrials.eu/trial/study-on-mrna-3705-for-patients-with-methylmalonic-acidemia-due-to-methylmalonyl-coa-mutase-deficiency/ Wed, 29 Apr 2026 14:28:59 +0000 https://clinicaltrials.eu/trial/study-on-mrna-3705-for-patients-with-methylmalonic-acidemia-due-to-methylmalonyl-coa-mutase-deficiency/ This clinical trial is focused on studying a rare genetic condition called Isolated Methylmalonic Acidemia (MMA), which is caused by a deficiency in an enzyme known as Methylmalonyl-CoA Mutase. This enzyme deficiency leads to the accumulation of certain substances in the body, which can cause serious health problems. The study is testing a new treatment called mRNA-3705, which is designed to help the body produce the missing enzyme. This treatment is given as an injection into a vein, which is known as an intravenous injection.

The purpose of the study is to evaluate the safety and effectiveness of mRNA-3705 in people with MMA. The study is divided into two parts. In the first part, researchers will focus on understanding how safe and tolerable the treatment is for participants. In the second part, they will look at how well the treatment works by comparing the frequency of metabolic decompensation events (MDEs), which are episodes when the body cannot properly process certain substances, with the standard care currently available.

Participants in the study will receive the treatment and be monitored over time to see how their bodies respond. The study will also measure changes in the levels of certain substances in the blood, as well as any side effects that may occur. This research aims to provide valuable information that could lead to better treatment options for people living with Isolated Methylmalonic Acidemia.

]]> Study on Long-Term Safety of mRNA-3705 for Patients with Methylmalonic Acidemia (MMA) https://clinicaltrials.eu/trial/study-on-long-term-safety-of-mrna-3705-for-patients-with-methylmalonic-acidemia-mma/ Wed, 29 Apr 2026 14:26:52 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-of-mrna-3705-for-patients-with-methylmalonic-acidemia-mma/ This clinical trial is focused on studying the long-term safety and effects of a treatment called mRNA-3705 in individuals with a rare genetic condition known as methylmalonic acidemia (MMA). This condition is caused by a deficiency in an enzyme called methylmalonyl-coenzyme A mutase, which is important for breaking down certain proteins and fats in the body. The study involves participants who have previously been part of other clinical trials involving mRNA-3705.

The purpose of this study is to evaluate how safe mRNA-3705 is when used over a longer period. Participants will receive the treatment through an intravenous infusion, which means it is delivered directly into the bloodstream. The study will monitor participants for any side effects and changes in their condition over time. This includes looking at levels of certain substances in the blood that are related to MMA, as well as any changes in the frequency of hospital visits or the impact of the disease on daily activities like school or work.

In addition to mRNA-3705, the study may involve other medications such as dexamethasone, paracetamol, cetirizine dihydrochloride, ibuprofen, and famotidine, which are used to manage symptoms or support the treatment process. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the treatment. The study is expected to continue until January 2029, allowing researchers to gather comprehensive data on the long-term safety and effectiveness of mRNA-3705 in managing MMA.

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