Propionic AcidemiaPhase 1/2NCT05130437
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
A Phase 1/2 study testing mRNA-3927 in people with Propionic Acidemia, run by ModernaTX, Inc. at 22 sites in 8 countries.
Trial summary
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Propionic Acidemia
- What is being tested?
- mRNA-3927
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 22 sitesin 8 countries
- Study dates
- 9 Nov 2021to 4 Dec 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participated in Study mRNA-3927-P101.
- Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.
Who cannot join
- Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
- Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
- History of liver and/or kidney transplant.
Trial locations and contacts
22 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
22 sites in 8 countries
United States7 sites, 7 recruiting
- Recruiting
University of Michigan Hospitals
Ann Arbor, Michigan, 48109
- Recruiting
Duke University Medical System (Duke Health)
Durham, North Carolina, 27710
- Recruiting
Texas Children's Hospital
Houston, Texas, 77030
- Not yet recruiting
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095
- Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
- Recruiting
University of Stanford Medical Center
Palo Alto, California, 94304-1503
- Recruiting
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
United Kingdom4 sites, 3 recruiting
- Recruiting
University Hospital Birmingham NHS Foundation Trust
Birmingham, West Midlands, B15 2TH
- Completed
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, West Midlands, B4 6NH
- Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
- Recruiting
Willink Biochemical Genetics Unit - Manchester
Manchester, England, M13 9WL
France2 sites, 2 recruiting
- Not yet recruiting
AP-HM- Hôpital de La Timone
Marseille, 13005
- Not yet recruiting
Hôpital Necker - Enfants Malades
Paris, 75019
Japan2 sites, 2 recruiting
- Recruiting
Tohoku University Hospital
Sendai, Miyagi, 980-8574
- Recruiting
Fujita Health University Hospital
Toyoake-shi, Akita, 470-1192
Netherlands2 sites, 2 recruiting
- Not yet recruiting
Erasmus MC -Dr. Molewaterplein 40
Rotterdam, South Holland, 3015 GD
- Not yet recruiting
Universitair Medisch Centrum Utrecht - PPDS
Utrecht, 3584 CX
Saudi Arabia2 sites, 2 recruiting
- Not yet recruiting
King Faisal Specialist Hospital & Research Centre
Riyadh, Ar Riya, 11211
- Not yet recruiting
King Abdullah Children's Specialist Hospital
Riyadh, Ar Riya, 14611
Spain2 sites, 2 recruiting
- Not yet recruiting
Hospital Universitario Cruces
Barakaldo, Vizcaya, 48903
- Not yet recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
Canada1 site, 1 recruiting
- Recruiting
Hospital For Sick Children
Toronto, Ontario, M5G 1X8
About this study
Official title: A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
Read the detailed description
The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.
The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.
- Conditions
- Propionic Acidemia
- Being tested
- mRNA-3927
- Keywords
- mRNA-3927, Propionic Aciduria, Metabolism, Inborn Errors, Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases, Organic Acidemias, Moderna, mRNA
- Medical subject headings
- Propionic Acidemia, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mRNA-3927Biological
mRNA-3927 dispersion for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| mRNA-3927 | Experimental | Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation. Receives: Biological: mRNA-3927 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
Measured: Baseline through End of Study Visit (up to 8 years)
Secondary outcomes (4)
Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)
Measured: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported MDE-related Hospitalizations
Measured: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Hospitalizations
Measured: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters
Measured: Baseline through End of Study Visit (up to 8 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2021
- Study start9 Nov 2021
- Primary completion4 Dec 2029 (estimated)
- Study completion4 Dec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Propionic AcidemiaStudies of the same condition, open and under way.
- More trials run by ModernaTX, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ModernaTX, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05130437
- Sponsor's protocol ID
- mRNA-3927-P101-EXT
- Other IDs
- 2022-502911-12-00
- Status last verified
- Mar 2026
- First submitted
- 3 Nov 2021
- Last updated
- 23 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
- ClinicalTrials.gov ID
- NCT05130437
- Acronym / sponsor ID
- mRNA-3927-P101-EXT
- Conditions
- Propionic Acidemia
- Keywords
- mRNA-3927, Propionic Aciduria, Metabolism, Inborn Errors, Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- ModernaTX, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Nov 2021 (actual)
- Primary completion
- 4 Dec 2029 (estimated)
- Study completion
- 4 Dec 2031 (estimated)
- First posted
- 23 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
Detailed description
The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.
The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mRNA-3927 | Experimental | Biological: mRNA-3927Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Participated in Study mRNA-3927-P101.
- Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.
Key exclusion criteria (3 listed)
- Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
- Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
- History of liver and/or kidney transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mRNA-3927mRNA-3927 dispersion for IV infusion | Biological | mRNA-3927 |
Primary outcome
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
Time frame: Baseline through End of Study Visit (up to 8 years)
4 secondary outcomes
Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported MDE-related Hospitalizations
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Hospitalizations
Time frame: Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters
Time frame: Baseline through End of Study Visit (up to 8 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
22 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing mRNA-3927 in people with Propionic Acidemia, run by ModernaTX, Inc. at 22 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, United Kingdom, France, Japan, Netherlands and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Moderna WeCare Team (1-866-663-3762, WeCareClinicalTrials@modernatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation (time frame: Baseline through End of Study Visit (up to 8 years)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2026.