RecruitingPHASE1, PHASE2INTERVENTIONAL

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

This study is an extension of previous research into a medicine called mRNA-3705, which is being investigated for a condition called Methylmalonic Acidemia (MMA). MMA is a rare genetic disorder where the body has trouble breaking down certain proteins and fats, leading to a build-up of harmful substances. The main goal of this extension study is to monitor the long-term safety and how well mRNA-3705 continues to work in people who have already received it in earlier studies. It’s important to understand the effects of new treatments over a longer period. Participants will continue to receive the medicine and be regularly checked by doctors, to ensure it's still safe and helpful. This study is for people who have already been taking part in initial trials for mRNA-3705.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

ModernaTX, Inc.

Enrolment target

Start

08 Mar 2022

Estimated completion

02 Apr 2034

Methylmalonic Acidemia

What is this study about?

You're looking at information about a clinical trial for a medicine called mRNA-3705. This medicine is being developed to treat a rare genetic condition called Methylmalonic Acidemia, often shortened to MMA. People with MMA have a problem breaking down certain parts of food, which can lead to harmful substances building up in their body.

This particular study is an 'extension study'. This means it's for people who have already taken part in earlier studies testing mRNA-3705. The main reason for this extension is to see how safe the medicine is and how well it works over a longer period. When a new medicine is developed, doctors and scientists first need to make sure it's safe and effective in the short term. Then, they need to check its long-term effects. This study helps gather that important information.

If you join, you would continue to receive mRNA-3705. The study team would carefully monitor your health and how your body responds to the treatment. This helps us understand if the medicine continues to be helpful and safe over time. The study will have a 'Treatment Period' where you receive the medicine, followed by a 'Follow-up Period' to monitor your health for a few months after your last dose. This careful monitoring is crucial for developing new and effective treatments for conditions like MMA.

Key takeaways

This study is for people already taking mRNA-3705 for Methylmalonic Acidemia (MMA).
Its main goal is to check the medicine's long-term safety and effectiveness.
Participation involves continuing the medicine and regular health check-ups.
The study continues until the medicine is available, or you stop, or your doctor advises otherwise.
You can leave the study at any time without affecting your normal care.

Who may be eligible?

This study is specifically designed for people who have already participated in other clinical studies involving mRNA-3705 for Methylmalonic Acidemia. Essentially, you need to have completed the main part of a previous study with this medicine, or have a specific reason (like a COVID-19 vaccine interruption) that allowed you to move to this study early from a previous one.

To be eligible, you must have finished your treatment time in a previous mRNA-3705 study. Also, your final check-up from that previous study must have happened within 10 days of your first dose in this new extension study. This ensures the study team has up-to-date information about your health.

However, there are also some reasons why you might not be able to join. For example, if the study doctor believes that continuing with mRNA-3705 wouldn't be helpful for your specific condition, or if you have other health issues (especially related to your liver or kidneys) that the doctor thinks could make taking part in the study risky for you. If you've had a liver or kidney transplant, you also wouldn't be able to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have I already taken part in a previous study for mRNA-3705 for MMA?
Did I complete the main treatment period of that previous study?
Is my final check-up from the last study within 10 days of starting this one?
Do I have any liver or kidney transplants?
Has my doctor said they believe this treatment would still be helpful for me?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would continue to receive the study medicine, mRNA-3705. The study is divided into two main parts: a 'Treatment Period' and a 'Follow-up Period'. During the Treatment Period, you'd regularly receive the medication. This period will continue until one of a few things happens: either the medicine officially becomes available to buy and is reimbursed in your country, or you decide to stop taking the medicine, or your doctor feels it's no longer benefiting you, or the company stops developing the medicine.

Once the Treatment Period ends, there will be a 'Follow-up Period' of up to six months after your last dose of mRNA-3705. During both periods, you would have regular visits and assessments with the study team. These check-ups are important to monitor your health, how you're responding to the treatment, and ensure your safety. They will involve various tests and discussions with the doctors and nurses. The total duration of your participation would depend on how long you remain in the Treatment Period, plus the additional six months of follow-up.

Potential risks and benefits

Participating in this study might mean you continue to experience potential benefits from mRNA-3705 if it's working for your condition, and it helps researchers learn more about the long-term effects of this new treatment. As with any medicine, there are potential risks and side effects, and these will be carefully monitored throughout the study. The study doctors will discuss all known potential risks with you in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care. The study team will always prioritise your well-being and safety.

Locations (12)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UCLA Medical Center
Verified postcode
Los Angeles, United States· Recruiting
Lucile Packard Children's Hospital at Stanford
Verified postcode
Palo Alto, United States· Recruiting
Altman Clinical and Translational Research Institution
Verified postcode
San Diego, United States· Recruiting
Stollery Children's Hospital University of Alberta
Verified postcode
Edmonton, Canada· Recruiting
Hospital For Sick Children
Verified postcode
Toronto, Canada· Recruiting
Hôpital Necker - Enfants Malades APHP
Verified postcode
Paris, France· Recruiting
Erasmus MC
Verified postcode
Rotterdam, Netherlands· Recruiting
Universitair Medisch Centrum Utrecht
Verified postcode
Utrecht, Netherlands· Recruiting
Hospital Universitario Cruces
Verified postcode
Barakaldo, Spain· Recruiting
Hospital Universitario 12 de Octubre
Verified postcode
Madrid, Spain· Recruiting
Birmingham Children's Hospital NHS Foundation Trust
Verified postcode
Birmingham, United Kingdom· Terminated
Royal Manchester Childrens Hospital
Verified postcode
Manchester, United Kingdom· Recruiting

Common questions

What is Methylmalonic Acidemia (MMA)?

MMA is a rare genetic condition where your body has trouble processing certain parts of food, leading to a build-up of harmful substances.

Why is this an 'extension study'?

It's an extension because it's for people who have already taken part in earlier studies of mRNA-3705. We want to see how the medicine works and if it's safe over a longer time.

How long will I be in the study?

The 'Treatment Period' where you receive the medicine continues as long as certain conditions are met, followed by up to 6 months of follow-up after your last dose.

Can I stop taking part if I want to?

Yes, you have the right to withdraw from the study at any time, for any reason, and it won't affect your regular medical care.

Will I have to pay for the medicine?

No, all study medicines and study-related care are provided free of charge.

How to find out more

Moderna WeCare Team

wecareclinicaltrials@modernatx.com 1-866-663-3762 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.