Propionic AcidemiaNCT05769621
A Retrospective Study to Characterize Participants With Propionic Acidemia
An observational study of Propionic Acidemia, run by ModernaTX, Inc. at 29 sites in 9 countries.
Trial summary
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Propionic Acidemia
- How many people
- 60 participants
- Where
- 29 sitesin 9 countries
- Study dates
- 16 Jun 2023to 30 Sep 2026
- Phase
- Observational
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals of a minimum age of 2 years with confirmed diagnosis of PA.
Who may be able to join
- Participants' medical records are eligible for abstraction only if all of the following criteria apply:
- Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunitα[PCCA] and/or propionyl-CoA carboxylase subunitβ[PCCB] mutations).
- Participant provided informed consent (and assent, as applicable).
- Medical records with sufficient data to support medical encounter for CEC adjudication (MEA adjudication) must be available dating back to birth or 01 January 2015, whichever occurs later. Participants born prior to 2015 will require additional information to confirm eligibility.
- Experienced at least one MDE in the 24 months preceding the Index Date or experienced at least 3 MDEs within any one 12-month retrospective review period (based on Index Date) dating back to birth or 01 January 2015, whichever occurs later.
- Must have a confirmed age (months) of disease onset (early or late, where early is defined as the neonatal period).
Show all 14Show fewer
- Participants who meet any of the censoring criteria listed below may be enrolled into the study if they have a minimum of 2 years of data available for abstraction prior to meeting the censoring criteria and they meet all inclusion criteria based on the data available for abstraction.
- Censoring Criteria:
- Participants medical records meeting the following censoring criterion will not be abstracted:
- Participation in a clinical study of any investigational agent.
- Received gene therapy treatment
- Confirmed organ transplantation
- Investigator is no longer able to obtain relevant clinical information of the participant.
- Death
Who cannot join
- Participant's medical records will not be eligible for abstraction if the participant meets the following exclusion criteria:
- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).
Trial locations and contacts
29 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
29 sites in 9 countries
Spain7 sites, 6 recruiting
- Recruiting
Hospital Universitario Cruces
Barakaldo, 48903
- Recruiting
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 8950
- Recruiting
Hospital Universitario Raymon y Cajal
Madrid, 28034
- Active, not recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
- Recruiting
Hospital Universitario Marques de Valdecilla
Santander, Cantabria, 39008
- Recruiting
Hospital Universitario Virgen del Rocio - PPDS
Seville, 41013
- Recruiting
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, 46026
United States6 sites, 4 recruiting
- Active, not recruiting
University of Michigan Hospital
Ann Arbor, Michigan, 48109-5000
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21287-0010
- Terminated
Texas Children's Hospital
Houston, Texas, 77030-2614
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105-3901
- Not yet recruiting
University of Stanford Medical Center
Stanford, California, 94305-2200
France4 sites, 3 recruiting
- Recruiting
CHU de Marseille - Hôpital de la Timone
Marseille, 13386
- Recruiting
Hôpital Necker - Enfants Malades
Paris, Paris, 75743
- Recruiting
CHU Toulouse
Toulouse, Haute-Garonne, 31300
- Withdrawn
CHRU Nancy Hôpital des Enfants
Vandœuvre-lès-Nancy, Meurthe-et-Moselle, 54511
United Kingdom4 sites, 1 recruiting
- Recruiting
University Hospital Birmingham
Birmingham, B29 6JD
- Active, not recruiting
Great Ormond Street Hospital
City of London, London
- Withdrawn
Victoria Hospital
Kirkcaldy, Fife, KY2 5AH
- Active, not recruiting
St Mary's Hospital - PPDS
Manchester, Lancashire
Italy3 sites, 2 recruiting
- Recruiting
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany, 50139
- Active, not recruiting
ASST di Monza - Azienda Ospedaliera San Gerardo
Monza, Monza A Brianza, 20900
- Recruiting
Azienda Ospedaliera Universitaria Federico II
Naples, Campania, 80131
Japan2 sites, 2 recruiting
- Not yet recruiting
The Jikei University School of Medicine
Minato, Tokyo, 105-8471
- Not yet recruiting
Fujita Health University Hospital
Toyoake, Aichi-ken, 470-1192
Australia1 site
- Withdrawn
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Canada1 site
- Withdrawn
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2B7
Netherlands1 site, 1 recruiting
- Not yet recruiting
Erasmus MC
Rotterdam, South Holland, 3015 GD
About this study
Official title: A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Read the detailed description
Data will be abstracted through medical record review ensuring inclusion only of data from participants meeting all the inclusion criteria and none of the censoring criteria. Data will be collected retrospectively from study sites, including medical clinics, hospitals and academic medical centers.
- Conditions
- Propionic Acidemia
- Medical subject headings
- Propionic Acidemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| PA Participants | Participants with PA who meet all eligibility criteria for medical record abstraction. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs)
Measured: Up to 10 years
Secondary outcomes (4)
Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale
Measured: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Hospitalizations
Measured: Up to 10 years
Number of Annualized CEC-adjudicated MDE-related Hospitalizations
Measured: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2023
- Study start16 Jun 2023
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2026 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 60 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
- NCT05769621
- Sponsor's protocol ID
- mRNA-3927-P002
- Status last verified
- Feb 2026
- First submitted
- 3 Mar 2023
- Last updated
- 5 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
- ClinicalTrials.gov ID
- NCT05769621
- Acronym / sponsor ID
- mRNA-3927-P002
- Conditions
- Propionic Acidemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- ModernaTX, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Jun 2023 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 15 Mar 2023 (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.
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Detailed description
Data will be abstracted through medical record review ensuring inclusion only of data from participants meeting all the inclusion criteria and none of the censoring criteria. Data will be collected retrospectively from study sites, including medical clinics, hospitals and academic medical centers.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PA Participants | – | –Participants with PA who meet all eligibility criteria for medical record abstraction. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals of a minimum age of 2 years with confirmed diagnosis of PA.
Key inclusion criteria (14 listed)
- Participants' medical records are eligible for abstraction only if all of the following criteria apply:
- Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunitα[PCCA] and/or propionyl-CoA carboxylase subunitβ[PCCB] mutations).
- Participant provided informed consent (and assent, as applicable).
- Medical records with sufficient data to support medical encounter for CEC adjudication (MEA adjudication) must be available dating back to birth or 01 January 2015, whichever occurs later. Participants born prior to 2015 will require additional information to confirm eligibility.
- Experienced at least one MDE in the 24 months preceding the Index Date or experienced at least 3 MDEs within any one 12-month retrospective review period (based on Index Date) dating back to birth or 01 January 2015, whichever occurs later.
Key exclusion criteria (2 listed)
- Participant's medical records will not be eligible for abstraction if the participant meets the following exclusion criteria:
- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs)
Time frame: Up to 10 years
4 secondary outcomes
Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Hospitalizations
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated MDE-related Hospitalizations
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits
Time frame: Up to 10 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 5 Feb 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.
29 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Propionic Acidemia, run by ModernaTX, Inc. at 29 sites in 9 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 14 inclusion and 2 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 5 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in Spain, United States, France, United Kingdom, Italy and 4 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.
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 Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) (time frame: Up to 10 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 5 Feb 2026.