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

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Administration of medication

    The medication mRNA-3927 is administered through an intravenous method, which means it is delivered directly into a vein using a needle or catheter.

    The medication mRNA-3927 is a dispersion for injection containing modified messenger ribonucleic acid (a type of genetic material) encapsulated into small fat particles called lipid nanoparticles.

  2. Step 2

    Monitoring and observation

    The focus of this stage is to monitor the long-term safety of the medication for individuals with propionic acidemia, a rare metabolic condition.

    Observations are made regarding the occurrence and seriousness of any adverse events, which are any side effects or unintended medical occurrences.

    The frequency of metabolic decompensation episodes, which are sudden worsening of symptoms related to the condition, is tracked.

    The frequency of hospitalizations and urgent healthcare encounters specifically related to propionic acidemia is recorded.

Who can join the trial?

7 criteria

  • 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 trial?

5 criteria

  • 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.
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Investigated drugs

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.

What is already known about the treatment

mRNA-3927 - This medication is an experimental treatment given as an injection directly into a vein to help people with a rare metabolic disorder called propionic acidemia. It works by using tiny fat bubbles to deliver genetic instructions into the body, which teaches cells to produce the specific proteins needed to break down certain fats and proteins correctly. Currently, this substance is being studied in clinical trials to see if it can safely and effectively help the body function better. It is classified as an mRNA-based therapy designed to replace missing or faulty proteins at the molecular level.

Investigated diseases

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 detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2022-502911-12-00Protocol codemRNA-3927-P101-EXTEstimated enrolment38 patientsSponsorModerna Therapeutics Inc.

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