Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

No treatment, just data: new study tracks rare metabolic disease

NCT ID NCT05769621

Knowledge-focused Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is not testing any new drug or treatment. Instead, researchers will look back at medical records of 60 people with propionic acidemia, a rare genetic disorder that affects how the body breaks down certain proteins and fats. The goal is to better understand the disease, including how often patients have serious metabolic episodes and need hospital care. This information could help design future studies and improve care.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Sep 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Individuals of a minimum age of 2 years with confirmed diagnosis of PA.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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).

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Propionic acidemia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

propionic acidemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Propionic acidemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    19 sites in 7 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • ASST di Monza - Azienda Ospedaliera San Gerardo

    ACTIVE_NOT_RECRUITING

    Monza, Monza A Brianza, 20900, Italy

  • Azienda Ospedaliera Universitaria Federico II

    RECRUITING

    Naples, Campania, 80131, Italy

  • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

    RECRUITING

    Florence, Tuscany, 50139, Italy

  • CHRU Nancy Hôpital des Enfants

    WITHDRAWN

    Vandœuvre-lès-Nancy, Meurthe-et-Moselle, 54511, France

  • CHU Toulouse

    RECRUITING

    Toulouse, Haute-Garonne, 31300, France

  • CHU de Marseille - Hôpital de la Timone

    RECRUITING

    Marseille, 13386, France

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Erasmus MC

    NOT_YET_RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Fujita Health University Hospital

    NOT_YET_RECRUITING

    Toyoake, Aichi-ken, 470-1192, Japan

  • Great Ormond Street Hospital

    ACTIVE_NOT_RECRUITING

    City of London, London, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Esplugues de Llobregat, Barcelona, 8950, Spain

  • Hospital Universitari I Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

  • Hospital Universitario 12 de Octubre

    ACTIVE_NOT_RECRUITING

    Madrid, 28041, Spain

  • Hospital Universitario Cruces

    RECRUITING

    Barakaldo, 48903, Spain

  • Hospital Universitario Marques de Valdecilla

    RECRUITING

    Santander, Cantabria, 39008, Spain

  • Hospital Universitario Raymon y Cajal

    RECRUITING

    Madrid, 28034, Spain

  • Hospital Universitario Virgen del Rocio - PPDS

    RECRUITING

    Seville, 41013, Spain

  • Hôpital Necker - Enfants Malades

    RECRUITING

    Paris, Paris, 75743, France

  • Johns Hopkins University

    RECRUITING

    Baltimore, Maryland, 21287-0010, United States

  • Royal Children's Hospital Melbourne

    WITHDRAWN

    Parkville, Victoria, 3052, Australia

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105-3901, United States

  • St Mary's Hospital - PPDS

    ACTIVE_NOT_RECRUITING

    Manchester, Lancashire, United Kingdom

  • Stollery Children's Hospital University of Alberta

    WITHDRAWN

    Edmonton, Alberta, T6G 2B7, Canada

  • Texas Children's Hospital

    TERMINATED

    Houston, Texas, 77030-2614, United States

  • The Jikei University School of Medicine

    NOT_YET_RECRUITING

    Minato, Tokyo, 105-8471, Japan

  • University Hospital Birmingham

    RECRUITING

    Birmingham, B29 6JD, United Kingdom

  • University of Michigan Hospital

    ACTIVE_NOT_RECRUITING

    Ann Arbor, Michigan, 48109-5000, United States

  • University of Stanford Medical Center

    NOT_YET_RECRUITING

    Stanford, California, 94305-2200, United States

  • Victoria Hospital

    WITHDRAWN

    Kirkcaldy, Fife, KY2 5AH, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.