At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 39 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks back at the medical records of people with propionic acidemia, a rare inherited condition, to learn more about their illness and care. It is run by ModernaTX, Inc. No new treatment is given.
Who can take part
- Diagnosis of propionic acidemia confirmed by genetic testing
- Consent given for the person's medical records to be used
- Medical records with enough information, going back to birth or 1 January 2015 (whichever is later)
- At least one major disease episode (MDE) in the 24 months before the index date, or at least 3 MDEs in any 12-month look-back period
- A known age (in months) when the disease started
- People who left the study early (for example, through death or a transplant) can still be included if they have at least 2 years of data and meet all other criteria
Who may not be able to
- Took part in a clinical study of any investigational (experimental) medicine
- Received gene therapy
- Had an organ transplant
- The researcher can no longer get the person's clinical information
- The person has died (records not abstracted on this basis)
- Taking part now or in the past in studies mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437)
What taking part involves
- • No treatment is given. Researchers review existing medical records to collect information about the condition.
Time commitment: Not stated — ask the trial team about visits, duration, and what taking part involves beyond allowing your medical records to be reviewed.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 2 Years and over
- Who
- All
- Number of participants
- 60
- Started
- 2023-06-16
- Last checked
- 2026-02
Plain English Summary
What is this study?
- • Testing a new treatment for propionic acidemia
- • Clinical study - 60 participants
- • This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA)
Who can take part?
- • Ages 2 Years and over
- • Diagnosed with propionic acidemia
Where?
- • Kirkcaldy - Victoria Hospital
- • Manchester - St Mary's Hospital - PPDS
- • City of London - Great Ormond Street Hospital
- • Birmingham - University Hospital Birmingham
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years and over
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ additional information to confirm eligibility
What the study is looking for
- ✓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...
- ✓Participant provided agreement to take part (and assent, as applicable).
- ✓Must have a confirmed age (months) of disease onset (early or late, where early is defined as the neonatal period).
- ✓Censoring Criteria:
Who cannot take part
- ✗Participant's medical records will not be eligible for abstraction if the participant meets the following exclusion...
- ✗\- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).
See the full criteria
Where Is This Study? (4 UK sites)
Victoria Hospital
Kirkcaldy KY2 5AH, United Kingdom
St Mary's Hospital - PPDS
Manchester, United Kingdom
Great Ormond Street Hospital
City of London, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700University Hospital Birmingham
Birmingham B29 6JD, United Kingdom
How to Get in Touch
Moderna WeCare Team
Sponsor contactCONTACT
