A study to evaluate the long-term safety and effectiveness of mRNA-3927 in patients with Propionic Acidemia

1 1 1

What is this study about?

This study focuses on Propionic Acidemia, which is a rare genetic disorder that prevents the body from properly breaking down certain proteins. This condition can lead to a buildup of acid in the blood, which may cause health problems. The study aims to evaluate the long-term safety and clinical activity of mRNA-3927, an experimental treatment made of modified messenger ribonucleic acid. This substance is delivered through intravenous injection, which means it is administered directly into a vein, and it is packaged in lipid nanoparticles, which are tiny fat-based particles used to protect the medicine as it enters the body.

Participants in this study have previously been part of an earlier clinical trial. During this phase, they will continue to receive the treatment to monitor how the body reacts over a longer period. The focus is on tracking any side effects or changes in health to ensure the treatment remains safe for long-term use.

Who Can Join the Study?

  • You must have participated in the previous study known as mRNA-3927-P101.
  • You must have finished the end-of-treatment (the final scheduled visit after finishing the medicine) for the previous study.
  • The first dose of the new medicine must be given between 11 and 17 days after your last dose in the previous study.
  • You or your legally authorized representative (a person allowed by law to make medical decisions for you, such as a parent or guardian) must agree to participate by signing an informed consent form, which is a document explaining all the details and risks of the study.
  • You or your caregiver (a person who helps look after you) must be willing and able to follow all the tests and checks required by the study.
  • The study is open to both males and females.
  • The study is open to individuals in the age range of 2 to 3 years old.

Who Cannot Join the Study?

  • If the doctor believes that continuing the mRNA-3927 medicine will not provide any helpful health benefits.
  • If you have any medical condition or unusual laboratory results (test results from your blood or other fluids) that make the doctor think participating in the study might be unsafe for you.
  • If you have a history of receiving a liver transplant (a surgery to replace a damaged liver with a healthy one) or a kidney transplant (a surgery to replace damaged kidneys with healthy ones).
  • If you are currently pregnant or breastfeeding.
  • If you are sexually active and do not agree to use a very reliable form of contraception (methods used to prevent pregnancy, such as birth control) during the study and for 3 months after your final dose.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Hospital Universitario 12 De Octubre Madrid Spain
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Hospital Universitario De Cruces Barakaldo Spain
Hopital Beaujon Clichy France
Apvkbknucn Pugxpvwd Heyglgrv Do Mvtjvfhkx Marseille France
Enpvzbh Urtljxaceugk Mggjakg Cuvvcbr Rfnyrfqds (ladenmg Muy 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
01.06.2026
Spain Spain
Recruiting
01.06.2026
The Netherlands The Netherlands
Not yet recruiting
01.06.2026

Trial locations

mRNA-3927 is a therapy that uses messenger RNA (mRNA) to help the body produce specific proteins needed for normal metabolism. In this study, it is given through an IV (intravenous) injection to help people with Propionic Acidemia (PA) manage their condition by providing the instructions cells need to function correctly.

Propionic acidemia – This is a metabolic disorder caused by the body’s inability to properly break down certain proteins and fats. It occurs due to a deficiency in an enzyme called propionyl-CoA carboxylase. As a result, propionic acid and other substances build up in the blood and tissues. This accumulation can lead to metabolic imbalances throughout the body. The condition typically begins shortly after birth or during infancy.

Trial ID:
2022-502911-12-00
Protocol code:
mRNA-3927-P101-EXT
NCT ID:
NCT05130437
Trial Phase:
Human Pharmacology (Phase I) – Other

Other Trials to Consider

  • Study on the Safety and Effectiveness of mRNA-3927 for Patients with Propionic Acidemia

    Recruiting

    2 1 1 1
    France Italy The Netherlands Spain