Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
This research study is looking at a new medicine called mRNA-3927 for a rare genetic condition called Propionic Acidemia (PA). The main goals are to check if the medicine is safe and how well people tolerate it. We also want to see if it helps improve the condition by looking at certain markers in the body. The study will happen in three main parts: first, finding the best dose for people aged one year and older; then, testing that dose more thoroughly in a larger group of people aged one year and older to understand its effectiveness; and finally, testing the medicine in babies under one year old. Participants will be carefully monitored throughout the study.
At a glance
What is this study about?
This study is about a new medicine called mRNA-3927 for a rare genetic condition called Propionic Acidemia, often shortened to PA. People with PA have trouble breaking down certain proteins and fats, which can lead to a build-up of harmful substances in the body. This can cause serious health problems, especially during metabolic crises.
The main goal of this research is to see if mRNA-3927 can help people with PA. We'll be looking carefully at how safe the medicine is and if it's well-tolerated by the body. We also want to understand how it works and whether it can improve the condition. Scientists will measure certain markers in the body to see if the medicine is having a positive effect. Ultimately, this study aims to find the right amount of medicine (the dose) that works best and is safest for patients.
This study is divided into three parts. The first part focuses on finding a safe and effective dose for people aged one year and older. Once we've found a good dose, the second part will involve more people from this age group to really understand how well the medicine works and its safety over a longer period. The third part of the study will then look at the safety and effects of mRNA-3927 specifically in babies who are less than one year old.
Key takeaways
- This study is testing a promising new medicine for Propionic Acidemia.
- It aims to find a safe and effective dose for a range of ages, from infants to adults.
- Participants will be closely monitored for safety and how well the medicine works.
- Participation involves observation, treatment with mRNA-3927, and follow-up.
- Joining could help advance understanding and treatment of Propionic Acidemia.
Who may be eligible?
To join this study, participants must have a confirmed diagnosis of Propionic Acidemia, which means it has been identified through a genetic test. For the first two people joining the study, they need to be at least 8 years old. After that, anyone joining must be at least 1 year old.
For babies under one year old, they can participate if they were identified with PA through newborn screening or have symptoms and a sibling with PA. They must also weigh at least 3 kilograms (about 6.6 pounds) and have had at least one PA-related health event, such as vomiting, not feeding well, breathing difficulties, or specific blood test abnormalities like metabolic acidosis or high ammonia levels.
Certain conditions would prevent someone from joining the study. This includes any other health issues that, in the doctor's opinion, might make it difficult to understand the study results, or other specific health problems identified during the initial checks.
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.
- Do I have a confirmed diagnosis of Propionic Acidemia?
- Am I at least 1 year old, or an infant under 1 year with specific health events?
- Have I had a genetic test that confirms my Propionic Acidemia diagnosis?
- Am I generally healthy enough to participate, without other significant medical conditions?
- If under 1 year, do I weigh at least 3 kilograms and have I had a PA-related health event?
What does participation involve?
If you join the study, you'll first have a period where doctors observe your health without any new medicine. Then, you'll start receiving the study medicine, mRNA-3927, for a set period. Throughout this treatment phase, doctors will closely monitor your health, take measurements, and perform tests to see how you're responding to the medicine and if you're experiencing any side effects. After the treatment period ends, there will be a follow-up period to ensure your health remains stable after stopping the medicine. The total number of hospital visits and the exact duration of each period will vary, but you can expect regular check-ups and assessments throughout the study.
Potential risks and benefits
Locations (35)
- UCSD Altman Clinical and Transalational Research Institute BuildingVerified postcodeLos Angeles, United States· Not yet recruiting
- Ronald Reagan UCLA Medical CenterVerified postcodeLos Angeles, United States· Recruiting
- Lucile Packard Children's Hospital StanfordVerified postcodeStanford, United States· Recruiting
- Nicklaus Children's HospitalVerified postcodeMiami, United States· Not yet recruiting
- University of South Florida - 12901 Bruce B DownsVerified postcodeTampa, United States· Not yet recruiting
- Ann and Robert H Lurie Childrens Hospital of ChicagoVerified postcodeChicago, United States· Recruiting
- Johns Hopkins Hospital, Adult Outpatient Clinical Research UnitVerified postcodeBaltimore, United States· Completed
- Boston Children's HospitalVerified postcodeBoston, United States· Completed
- University of Michigan HospitalsVerified postcodeAnn Arbor, United States· Recruiting
- Icahn School of Medicine at Mount Sinai - Clinical Research UnitVerified postcodeNew York, United States· Recruiting
- Duke University Medical System (Duke Health)Verified postcodeDurham, United States· Recruiting
- Cincinnati Children's Hospital Medical CenterVerified postcodeCincinnati, United States· Completed
Common questions
What is Propionic Acidemia?
Propionic Acidemia (PA) is a rare inherited condition where the body can't properly process certain proteins and fats, leading to harmful substances building up in the blood.
What is mRNA-3927?
mRNA-3927 is a new experimental medicine being tested in this study to see if it can help treat Propionic Acidemia.
Who can join this study?
The study is for people with a confirmed diagnosis of Propionic Acidemia, including children aged one year and older, and infants under one year, who meet specific health criteria.
What does a 'Phase 1/2' study mean?
This means the study is in its early stages. Phase 1 focuses on finding a safe dose and understanding how the medicine works, while Phase 2 further evaluates its effectiveness and safety in a larger group.
Will I receive the new medicine if I join?
Yes, if you meet the study criteria and are enrolled, you will receive the investigational medicine, mRNA-3927, according to the study plan.
How to find out more
Moderna WeCare Team
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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