A Retrospective Study to Characterize Participants With Propionic Acidemia

NCT05769621 | Recruiting

• 1 other identifier: mRNA-3927-P002
• Study Type: observational
• Target: 60
• Locations: 9 countries
• Sites: 29

Brief Summary

This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).

Conditions

Propionic Acidemia

Outcome Measures

Primary Outcomes (1)

• Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs)

  Up to 10 years

Secondary Outcomes (4)

• Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale

  Up to 10 years

• Number of Annualized CEC-adjudicated PA-related Hospitalizations

  Up to 10 years

• Number of Annualized CEC-adjudicated MDE-related Hospitalizations

  Up to 10 years

• Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits

  Up to 10 years

Study Arms (1)

PA Participants

Participants with PA who meet all eligibility criteria for medical record abstraction.

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• 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).
• 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

You may not qualify if:

• \- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).

Sponsors & Collaborators

• ModernaTX, Inc.: lead

Study Sites (29)

University of Stanford Medical Center

Stanford, California, 94305-2200, United States

NOT YET RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287-0010, United States

RECRUITING

University of Michigan Hospital

Ann Arbor, Michigan, 48109-5000, United States

ACTIVE NOT RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030-2614, United States

TERMINATED

Seattle Children's Hospital

Seattle, Washington, 98105-3901, United States

RECRUITING

Royal Children's Hospital Melbourne

Parkville, Victoria, 3052, Australia

WITHDRAWN

Stollery Children's Hospital University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

WITHDRAWN

CHU Toulouse

Toulouse, Haute-Garonne, 31300, France

RECRUITING

CHRU Nancy Hôpital des Enfants

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

WITHDRAWN

Hôpital Necker - Enfants Malades

Paris, Paris, 75743, France

RECRUITING

CHU de Marseille - Hôpital de la Timone

Marseille, 13386, France

RECRUITING

Azienda Ospedaliera Universitaria Federico II

Naples, Campania, 80131, Italy

RECRUITING

ASST di Monza - Azienda Ospedaliera San Gerardo

Monza, Monza A Brianza, 20900, Italy

ACTIVE NOT RECRUITING

Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

Florence, Tuscany, 50139, Italy

RECRUITING

Fujita Health University Hospital

Toyoake, Aichi-ken, 470-1192, Japan

NOT YET RECRUITING

The Jikei University School of Medicine

Minato, Tokyo, 105-8471, Japan

NOT YET RECRUITING

Erasmus MC

Rotterdam, South Holland, 3015 GD, Netherlands

NOT YET RECRUITING

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 8950, Spain

RECRUITING

Hospital Universitario Marques de Valdecilla

Santander, Cantabria, 39008, Spain

RECRUITING

Hospital Universitario Cruces

Barakaldo, 48903, Spain

RECRUITING

Hospital Universitario Raymon y Cajal

Madrid, 28034, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

ACTIVE NOT RECRUITING

Hospital Universitario Virgen del Rocio - PPDS

Seville, 41013, Spain

RECRUITING

Hospital Universitari I Politecnic La Fe de Valencia

Valencia, 46026, Spain

RECRUITING

Victoria Hospital

Kirkcaldy, Fife, KY2 5AH, United Kingdom

WITHDRAWN

St Mary's Hospital - PPDS

Manchester, Lancashire, United Kingdom

ACTIVE NOT RECRUITING

Great Ormond Street Hospital

City of London, London, United Kingdom

ACTIVE NOT RECRUITING

University Hospital Birmingham

Birmingham, B29 6JD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Propionic Acidemia

Condition Hierarchy (Ancestors)

Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Moderna WeCare Team

CONTACT

+1-866-663-3762; WeCareClinicalTrials@modernatx.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2023
• First Posted: March 15, 2023
• Study Start: June 16, 2023
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: February 5, 2026

Record last verified: 2026-02

Locations

GB (4); JP (2); IT (3); US (6); NL (1); ES (7); AU (1); CA (1); FR (4)