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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
Virgen del Rocío University Hospital	Sevilla	Spain
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Hospital Universitario De Cruces	Barakaldo	Spain
Hopital Beaujon	Clichy	France
UOC Malattie Metaboliche ed Epatologia	Rome	Italy
Avpzntsbcx Ppbdukep Hyxvlntl Dc Mktgmjait	Marseille	France
Evxehnt Uznlcduxjzaq Mhhhodb Ckulruh Rmcnqhpls (fxtjtum Mjh	Rotterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
France	Recruiting	01.01.2025
Italy	Not recruiting	01.01.2025
Spain	Not recruiting	01.01.2025
The Netherlands	Not recruiting	01.01.2025

Trial locations

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.

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 ID:

2022-502910-10-00

Protocol code:

mRNA-3927-P101

NCT ID:

NCT04159103

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?