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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Oncopole Claudius Regaud Toulouse France

Other Sites

Site Name City Country Status
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Hospital Universitario 12 De Octubre Madrid Spain
Virgen del Rocío University Hospital Sevilla Spain
Hospital Universitario De Cruces Barakaldo Spain
Universidade De Santiago De Compostela Santiago De Compostela Spain
Hopital Beaujon Clichy France
Ahhenuzdai Podxdnng Hebdpxej Dx Mdbsppzqi Marseille France
Etnceol Ukekgyqrvrzj Mggadfk Cehdebt Rxmbdezqb (kolspew Mwh Rotterdam The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
08.08.2023
Spain Spain
Recruiting
08.08.2023
The Netherlands The Netherlands
Recruiting
08.08.2023

Trial locations

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.

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.

Trial ID:
2022-501997-20-00
Protocol code:
mRNA-3705-P101-EXT
NCT ID:
NCT05295433
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

Other Trials to Consider

  • Study on mRNA-3705 for Patients with Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

    Not recruiting

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    France The Netherlands Spain