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

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a rare genetic condition called Propionic Acidemia. This condition affects the body's ability to break down certain proteins and fats, leading to a buildup of harmful substances. The study will use a new treatment called mRNA-3927, which is a type of gene therapy. This treatment involves using modified messenger ribonucleic acid (mRNA) to help the body produce specific proteins that are missing or not working properly in people with Propionic Acidemia. The mRNA is delivered into the body using tiny particles called lipid nanoparticles.

The purpose of the study is to evaluate the safety and effectiveness of mRNA-3927 in people with Propionic Acidemia. Participants will receive the treatment through an intravenous (IV) infusion, which means it will be administered directly into a vein. The study will be conducted in two parts. In the first part, the focus will be on assessing the safety and tolerability of the treatment. In the second part, the study will look at how well the treatment works by comparing the frequency of metabolic decompensation events (MDEs) in participants receiving the treatment to those receiving standard care.

Throughout the study, participants will be monitored for any side effects and changes in their condition. The study will also measure various biological markers in the blood to understand how the treatment affects the body. This trial aims to provide valuable information about the potential benefits and risks of using mRNA-3927 as a treatment for Propionic Acidemia, which could lead to new therapeutic options for individuals affected by this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on age and a diagnosis of propionic acidemia through genetic testing.

    Consent is required from the participant or their legal representative, and agreement to use effective contraception if applicable.

  2. Step 2

    Part 1: safety and tolerability assessment

    The initial phase focuses on evaluating the safety and tolerability of the investigational product, mRNA-3927.

    Participants receive mRNA-3927 through intravenous administration. The dosage and frequency are determined by the study protocol.

    Monitoring includes checking for any adverse effects and measuring specific blood levels before and after administration.

  3. Step 3

    Part 2: efficacy evaluation

    The second phase assesses the effectiveness of mRNA-3927 in reducing the frequency of metabolic decompensation events (MDEs).

    Participants continue to receive mRNA-3927 intravenously, with the dosage and frequency as per the study protocol.

    The study measures the annualized frequency of MDEs and other health indicators over a 12-month period.

  4. Step 4

    Ongoing monitoring and assessments

    Throughout the study, regular assessments are conducted to monitor health status and any side effects.

    Blood tests are performed to evaluate changes in specific biomarkers and to estimate pharmacokinetic parameters.

  5. Step 5

    Completion of study participation

    Upon completion of the study, participants undergo a final assessment to evaluate overall health and any long-term effects of the treatment.

    Participants are informed about the study findings and any implications for their health.

Who can join the trial?

6 criteria

  • Must be at least 8 years old at the time of giving consent if you are one of the first two participants in Part 1 of the study.
  • Must be at least 1 year old at the time of giving consent if you join after the first two participants.
  • Must have a confirmed diagnosis of **Propionic Acidemia (PA)**, which is a condition identified through specific genetic tests.
  • You or your legal representative must be willing and able to give informed consent, which means you understand the study and agree to participate, and you must be able to follow the study's assessments.
  • If you are a sexually active female who can have children or a sexually active male who can father children, you must agree to use a highly effective method of birth control during the study and for 3 months after the last dose of the study drug.
  • (For Part 2 only) Must have had at least one documented **Metabolic Decompensation Event (MDE)**, which is a specific health event related to PA, in the 12 months before giving consent.

Who cannot join the trial?

9 criteria

  • Individuals who do not have a confirmed diagnosis of Propionic Acidemia cannot participate. Propionic Acidemia is a rare genetic disorder that affects the body's ability to process certain parts of proteins and fats.
  • Participants who are not within the specified age range for the study are excluded. The study is open to certain age groups only.
  • Individuals who are not able to follow the study procedures or attend the required visits will not be able to participate.
  • Participants who have other medical conditions that might interfere with the study or pose a risk to their health during the study will be excluded.
  • Individuals who are currently participating in another clinical trial or have recently participated in one may not be eligible.
  • Pregnant or breastfeeding women are not allowed to participate in the study.
  • Participants who have a history of allergic reactions to similar treatments or components of the study medication will be excluded.
  • Individuals who have received certain medications or treatments that could affect the study results may not be eligible.
  • Participants who have a history of substance abuse or other behaviors that could interfere with the study will be excluded.
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Investigated drugs

mRNA-3927 is an investigational medication being studied for its potential to treat Propionic Acidemia, a rare genetic disorder. This medication is designed to provide the body with instructions to produce a specific protein that may be missing or not working properly in individuals with this condition. The study aims to assess the safety and how well the body tolerates this medication, as well as its effectiveness in reducing the frequency of metabolic decompensation events, which are serious episodes that can occur in people with Propionic Acidemia.

What is already known about the treatment

mRNA-3927 – This medication is administered through intravenous infusion, allowing it to be directly delivered into the bloodstream. Currently, mRNA-3927 is in the clinical trial phase, specifically a Phase 1/2 study, and is being investigated for its potential use in treating Propionic Acidemia, a rare metabolic disorder. The main therapeutic indication for mRNA-3927 is to address the underlying enzyme deficiency in Propionic Acidemia by providing the body with the necessary instructions to produce the missing or dysfunctional enzyme. At the molecular level, mRNA-3927 works by delivering synthetic messenger RNA (mRNA) into cells, which then instructs the cells to produce the enzyme that is deficient in individuals with this condition. It is classified under gene therapy medications, focusing on correcting genetic disorders by introducing functional genes.

Investigated diseases

Propionic Acidemia – Propionic Acidemia is a rare genetic disorder that affects the body's ability to break down certain parts of proteins and lipids. This condition is caused by a deficiency of the enzyme propionyl-CoA carboxylase, which leads to the accumulation of toxic substances in the blood. Symptoms often appear in early infancy and can include poor feeding, vomiting, low muscle tone, and lethargy. As the disease progresses, individuals may experience developmental delays and recurrent episodes of metabolic decompensation, which can lead to more severe symptoms. These episodes can be triggered by illness, fasting, or increased protein intake. Over time, the accumulation of toxic substances can affect various organs and systems in the body.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2022-502910-10-00Protocol codemRNA-3927-P101Estimated enrolment57 patientsSponsorModerna Therapeutics Inc.

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