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A Retrospective Study to Characterize Participants With Propionic Acidemia

Sponsor: ModernaTX, Inc.

NCT ID: NCT05769621

View on ClinicalTrials.gov ↗

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.

Propionic Acidemia

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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
Inclusion Criteria: 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). * 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 Exclusion Criteria: 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).

Where Is This Study? (4 UK sites)

Victoria Hospital

Kirkcaldy KY2 5AH, United Kingdom

WITHDRAWN
Hospital R&D contact (matched)

Sarah Dawe

sarah.dawe2@nhs.net01342 414573

St Mary's Hospital - PPDS

Manchester, United Kingdom

ACTIVE_NOT_RECRUITING

Great Ormond Street Hospital

City of London, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

University Hospital Birmingham

Birmingham B29 6JD, United Kingdom

Recruiting
Hospital R&D contact (matched)

Sarah Pountain Head of Research Governance

R&D@uhb.nhs.uk0121 371 4185

How to Get in Touch

Moderna WeCare Team

Sponsor contact

CONTACT

+1-866-663-3762 WeCareClinicalTrials@modernatx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-02