RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

This clinical trial is designed to check the ongoing safety and how well a treatment called mRNA-3927 works for people living with a rare genetic condition called propionic acidemia. All participants in this study would have already taken part in an earlier study for the same treatment. The main goal is to understand the long-term effects of mRNA-3927, meaning what happens when people use it for a longer period. Participants will continue to receive the treatment at the same dose they had in the previous study. There will be a treatment period, followed by a follow-up period to monitor their health. This helps doctors learn more about managing propionic acidemia safely and effectively.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

ModernaTX, Inc.

Enrolment target

Start

09 Nov 2021

Estimated completion

04 Dec 2031

Propionic Acidemia

What is this study about?

This research study is about a condition called propionic acidemia (PA). This is a rare, inherited condition where the body has trouble breaking down certain proteins and fats. This can lead to a build-up of harmful substances, which can cause serious health problems if not managed.

The study is testing a treatment called mRNA-3927. This particular study is an extension of an earlier study. This means it's for people who have already received mRNA-3927 in a previous trial. By continuing to follow these participants, the doctors can gather very important information on the long-term safety and effects of the treatment. This helps them understand how people cope with the treatment over a longer time and if it continues to be helpful for their condition.

Learning more about mRNA-3927 could potentially help people with propionic acidemia in the future by providing doctors with more information on how to treat this condition safely and effectively over the long term. This allows for better understanding and potential improvements in care.

Key takeaways

This study is an extension for people already on mRNA-3927 for propionic acidemia.
The main goal is to check the long-term safety and effects of the treatment.
Participants will continue with the same dose and schedule of mRNA-3927.
There's a treatment period, followed by a 3-month monitoring period.
It's open to eligible people aged 1 year and older who completed the previous study.

Who may be eligible?

To join this study, you must have already taken part in a previous study for the same treatment (called mRNA-3927-P101). You also need to have completed all the visits for that first study. Importantly, you would need to start this study very soon after finishing the last one, usually within about two weeks of your last dose of mRNA-3927.

There are also some reasons why you might not be able to join. For example, if the doctor doesn't think the treatment would still be helpful for you, or if there's any other health issue or test result that the doctor believes might put you at extra risk by being in the study.

Also, if you've had a liver or kidney transplant in the past, unfortunately, you wouldn't be able to join this particular study. The study is open to everyone aged 1 year and older, no matter their gender.

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.

Did I take part in the previous mRNA-3927-P101 study?
Did I complete all the visits for that first study?
Could I start this new study within about 2 weeks of my last dose of mRNA-3927?
Do I have any liver or kidney transplants in my medical history?
Am I aged 1 year or older?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will continue to receive the treatment called mRNA-3927. You'll receive the same dose and at the same schedule as you did in the previous study. The study involves two main parts: a 'Treatment Period' where you continue to get the medication, and then a 'Follow-up Period' which lasts for 90 days (about three months) after your last treatment dose.

During both periods, you will have regular visits to the clinic. These visits will involve various checks, such as blood tests, physical exams, and other assessments, to carefully monitor your health and how you are responding to the treatment. This is to ensure your safety and to see how the treatment is working over a longer time. The total duration of your participation will depend on how long you continue with the treatment and then the 90-day follow-up.

Potential risks and benefits

Taking part in a study like this means that doctors will be closely monitoring your health, which can be a benefit. The potential benefit of continuing the treatment is that it might continue to help manage your propionic acidemia. However, similar to all medicines, there can be potential risks or side effects from mRNA-3927. The research team will explain all known and potential side effects in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (22)

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

Ronald Reagan UCLA Medical Center
Verified postcode
Los Angeles, United States· Not yet recruiting
University of Stanford Medical Center
Verified postcode
Palo Alto, United States· Recruiting
University of Michigan Hospitals
Verified postcode
Ann Arbor, United States· Recruiting
Icahn School of Medicine at Mount Sinai
Verified postcode
New York, United States· Recruiting
Duke University Medical System (Duke Health)
Verified postcode
Durham, United States· Recruiting
The Children's Hospital of Philadelphia (CHOP)
Verified postcode
Philadelphia, United States· Recruiting
Texas Children's Hospital
Verified postcode
Houston, United States· Recruiting
Hospital For Sick Children
Verified postcode
Toronto, Canada· Recruiting
AP-HM- Hôpital de La Timone
Verified postcode
Marseille, France· Not yet recruiting
Hôpital Necker - Enfants Malades
Verified postcode
Paris, France· Not yet recruiting
Fujita Health University Hospital
Unverified
Toyoake-shi, Japan· Recruiting
Tohoku University Hospital
Verified postcode
Sendai, Japan· Recruiting

Common questions

What is propionic acidemia?

It's a rare inherited condition where your body can't properly break down certain proteins and fats, leading to harmful build-ups.

I was in the previous study (mRNA-3927-P101). Can I automatically join this one?

Not automatically. You need to meet all the eligibility rules for this new study, including timing and doctor's assessment.

Will I get a different dose of the medication?

No, you will receive mRNA-3927 at the same dose and schedule you had in the previous study.

How long will the study last for me?

It depends on how long you receive the treatment, plus a 90-day (3-month) follow-up period afterwards.

What if I decide I don't want to continue the study?

You can leave the study at any time, and it won't affect your regular medical care.

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.