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Study on Long-Term Safety of mRNA-3705 for Patients with Methylmalonic Acidemia (MMA)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
Receive the first dose of mRNA-3705 through an injection. This medication is designed to help manage isolated methylmalonic acidemia (MMA) due to a specific enzyme deficiency.
The injection is administered intravenously, meaning it is given directly into a vein.
Step 2
Ongoing treatment phase
Continue receiving mRNA-3705 injections at regular intervals as determined by the study protocol. The frequency and dosage will be specified by the healthcare team.
Monitor for any side effects or changes in health, and report these to the healthcare team.
Step 3
Supportive care
Take additional medications as needed to manage symptoms or side effects. These may include paracetamol for pain relief, fexofenadine hydrochloride or cetirizine dihydrochloride for allergies, and ibuprofen for inflammation.
These medications are typically taken orally, meaning they are swallowed in pill or liquid form.
Step 4
Regular assessments
Undergo regular health assessments to monitor the effectiveness of the treatment and any potential side effects.
These assessments may include blood tests to measure levels of methylmalonic acid and other biomarkers.
Step 5
Follow-up phase
Participate in follow-up visits to evaluate long-term safety and clinical activity of the treatment.
These visits will help determine the overall impact of the treatment on health and quality of life.

Who can join the trial?

7 criteria

Participants must have a condition called **isolated methylmalonic acidemia (MMA)** due to a deficiency in an enzyme known as **methylmalonyl-coenzyme A mutase (MUT)**.
Participants must have completed the treatment period in other clinical studies of **mRNA-3705** or be eligible to join this study early if they missed more than 3 doses of the study drug due to **COVID-19** vaccination during the previous study.
Participants must have completed the **End of Treatment (EOT) Visit** in the previous study within 10 days of receiving the first dose of **mRNA-3705** in the current study. This does not apply to those who received extra doses in the previous study because this study will not start open enrollment at the site by the time the treatment period is completed.
The participant or their legally authorized representative must be willing and able to provide **informed consent** or **assent** as required by local regulations. **Informed consent** means agreeing to participate in the study after understanding all the details, and **assent** is a similar agreement given by someone who is not legally able to give full consent, like a child.
The participant's caregiver, and the participant if they can give consent or assent, must be willing and able to follow the study-related assessments.
Participants can be of any gender.
The study includes vulnerable populations, which means it may involve people who need special protection or consideration.

Who cannot join the trial?

2 criteria

Participants who have not been diagnosed with **methylmalonic acidemia (MMA)** due to **methylmalonyl-coenzyme A mutase (MUT) deficiency**. This means that if you do not have this specific condition, you cannot join the study.
Participants who have not previously participated in other clinical studies of **mRNA-3705**. This means that if you have not been part of earlier studies involving this treatment, you cannot take part in this one.

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Verified sites

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Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands
Responsive
Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands

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Investigated drugs

mRNA-3705 is an investigational medication being studied for its long-term safety and clinical activity. It is designed for participants with a condition known as MMA, who have already taken part in previous clinical studies involving this medication. The trial aims to gather more information on how safe mRNA-3705 is for long-term use and how well it continues to work in these participants.

What is already known about the treatment

mRNA-3705 – This medication is administered through an injection, specifically designed for individuals with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency. Currently, mRNA-3705 is in the clinical trial phase, with ongoing studies evaluating its long-term safety and clinical activity. The main therapeutic indication is to address the metabolic disorder MMA, which is a rare genetic condition. At the molecular level, mRNA-3705 works by providing the necessary instructions to produce the deficient enzyme, thereby helping to correct the underlying enzyme deficiency. It is classified under gene therapy medications, focusing on delivering genetic material to treat or prevent disease.

Investigated diseases

Methylmalonic Acidemia – Methylmalonic acidemia (MMA) is a rare genetic disorder characterized by the body's inability to properly break down certain fats and proteins. This condition is caused by a deficiency in the enzyme methylmalonyl-coenzyme A mutase, which is crucial for metabolizing specific amino acids and lipids. As a result, toxic substances accumulate in the blood, leading to various symptoms. Individuals with MMA may experience developmental delays, feeding difficulties, and recurrent episodes of illness. Over time, the buildup of harmful substances can affect multiple organs and systems. The severity and progression of symptoms can vary widely among affected individuals.