Ongoing Clinical Trials for Methylmalonic Acidaemia
Methylmalonic acidaemia is a rare genetic disorder affecting the body’s ability to break down certain proteins and fats. Currently, there are 2 ongoing clinical trials testing a new treatment approach called mRNA-3705, which aims to help the body produce the missing enzyme. These studies are taking place in the Netherlands, France, and Spain, and are evaluating both the safety and effectiveness of this investigational therapy for patients with this condition.
Clinical trial locations
- France
- Netherlands
- Spain
Study on Long-Term Safety of mRNA-3705 for Patients with Methylmalonic Acidemia (MMA)
This trial is designed to evaluate the long-term safety of mRNA-3705 in patients who have already participated in earlier studies of this treatment. The investigational drug is delivered directly into the bloodstream through an intravenous infusion.
Who can participate:
- People diagnosed with isolated methylmalonic acidaemia caused by a deficiency in the methylmalonyl-coenzyme A mutase enzyme
- Those who have completed treatment in previous mRNA-3705 studies, or those who missed more than 3 doses in earlier studies due to COVID-19 vaccination
- Participants must have completed their end of treatment visit in the previous study within 10 days of receiving the first dose in this new study
- Participants or their legal representatives must be willing to provide informed consent and follow study assessments
- The study is open to all genders and includes vulnerable populations who may need special consideration
Who cannot participate:
- People who do not have methylmalonic acidaemia due to methylmalonyl-coenzyme A mutase deficiency
- Those who have not previously participated in other mRNA-3705 clinical studies
Study focus: The main goal is to monitor the long-term safety of mRNA-3705 and track any side effects over an extended period. Researchers will measure levels of certain substances in the blood related to the condition and observe changes in hospital visits and the impact on daily activities such as school or work. The study will continue until January 2029 to gather comprehensive safety data.
Investigational drug: mRNA-3705 is a gene therapy medication that provides instructions to the body to produce the missing enzyme. In addition to the main treatment, participants may receive supportive medications such as dexamethasone, paracetamol, cetirizine dihydrochloride, ibuprofen, and famotidine to manage symptoms. Some participants may receive a placebo for comparison purposes.
Study on mRNA-3705 for Patients with Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
This clinical trial investigates mRNA-3705 as a potential treatment for people with isolated methylmalonic acidaemia. The study is divided into two parts: the first focuses on safety and tolerability, while the second examines how well the treatment works compared to standard care.
Who can participate:
- Participants must be at least 1 year old and weigh at least 11 kilograms
- Must have a confirmed genetic diagnosis of isolated methylmalonic acidaemia due to methylmalonyl-CoA mutase deficiency
- Blood vitamin B12 levels must be within or above normal range (elevated levels are acceptable if due to B12 supplements)
- Participants or their legal representatives must be willing to give consent and follow study procedures
- Women of childbearing age and men who can father children must use effective birth control during the study and for 3 months after the last dose
- For Part 2 of the study, participants must have experienced at least one metabolic decompensation event in the 12 months before joining
Who cannot participate:
- People with other serious health conditions that could interfere with the study
- Those currently taking medications that might affect study results
- Individuals with recent infections or illnesses that could impact their health during the study
- People with a history of allergic reactions to similar treatments
- Women who are pregnant or breastfeeding
- Those who have recently participated in another clinical trial
- People unable to follow study procedures or attend required visits
Study focus: The trial aims to evaluate the safety and effectiveness of mRNA-3705 by measuring how often metabolic decompensation events occur compared to standard care. These events happen when the body cannot properly process certain substances. Researchers will also monitor blood levels of methylmalonic acid and other health indicators throughout the study, which is expected to conclude by May 2027.
Investigational drug: mRNA-3705 is administered through an intravenous infusion. It is a gene therapy product that delivers synthetic messenger RNA to cells, instructing them to produce the missing enzyme. This approach aims to correct the underlying metabolic problem by helping the body make the enzyme it lacks.
Summary
Both ongoing clinical trials are testing the same investigational treatment, mRNA-3705, in three European countries: the Netherlands, France, and Spain. This concentration of trials in Western Europe suggests a coordinated research effort to evaluate this new gene therapy approach.
One trial focuses specifically on long-term safety monitoring for patients who have already received mRNA-3705 in earlier studies, while the other is evaluating both initial safety and effectiveness in a broader patient population. Together, these studies provide a comprehensive assessment of this investigational therapy, from initial testing through long-term follow-up.
The focus on mRNA-3705 across both trials reflects the current research direction in this field, where gene therapy approaches are being explored as potential treatments for genetic enzyme deficiencies. The studies are designed to provide important information about whether this type of treatment can help manage the condition and improve quality of life for affected individuals.