A Retrospective Cohort Study to Characterize Propionic Acidemia Patients

Status: Recruiting
Location: See all (29) locations...
Study Type: Observational
SUMMARY

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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:

⁃ 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

Locations
United States
California
University of Stanford Medical Center
NOT_YET_RECRUITING
Stanford
Maryland
Johns Hopkins University
RECRUITING
Baltimore
Michigan
University of Michigan Hospital
ACTIVE_NOT_RECRUITING
Ann Arbor
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Texas
Texas Children's Hospital
TERMINATED
Houston
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Other Locations
Australia
Royal Children's Hospital Melbourne
WITHDRAWN
Parkville
Canada
Stollery Children's Hospital University of Alberta
WITHDRAWN
Edmonton
France
CHU de Marseille - Hôpital de la Timone
RECRUITING
Marseille
Hôpital Necker - Enfants Malades
RECRUITING
Paris
CHU Toulouse
RECRUITING
Toulouse
CHRU Nancy Hôpital des Enfants
WITHDRAWN
Vandœuvre-lès-nancy
Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
RECRUITING
Florence
ASST di Monza - Azienda Ospedaliera San Gerardo
ACTIVE_NOT_RECRUITING
Monza
Azienda Ospedaliera Universitaria Federico II
RECRUITING
Napoli
Japan
The Jikei University School of Medicine
NOT_YET_RECRUITING
Minato
Fujita Health University Hospital
NOT_YET_RECRUITING
Toyoake
Netherlands
Erasmus MC
NOT_YET_RECRUITING
Rotterdam
Spain
Hospital Universitario Cruces
RECRUITING
Barakaldo
Hospital Sant Joan de Deu
RECRUITING
Esplugues De Llobregat
Hospital Universitario 12 de Octubre
ACTIVE_NOT_RECRUITING
Madrid
Hospital Universitario Raymon y Cajal
RECRUITING
Madrid
Hospital Universitario Marques de Valdecilla
RECRUITING
Santander
Hospital Universitario Virgen del Rocio - PPDS
RECRUITING
Seville
Hospital Universitari I Politecnic La Fe de Valencia
RECRUITING
Valencia
United Kingdom
University Hospital Birmingham
RECRUITING
Birmingham
Great Ormond Street Hospital
ACTIVE_NOT_RECRUITING
City Of London
Victoria Hospital
WITHDRAWN
Kirkcaldy
St Mary's Hospital - PPDS
ACTIVE_NOT_RECRUITING
Manchester
Contact Information
Primary
Moderna WeCare Team
WeCareClinicalTrials@modernatx.com
+1-866-663-3762
Time Frame
Start Date: 2023-06-16
Estimated Completion Date: 2026-09-30
Participants
Target number of participants: 60
Treatments
PA Participants
Participants with PA who meet all eligibility criteria for medical record abstraction.
Related Therapeutic Areas
Sponsors
Leads: ModernaTX, Inc.

This content was sourced from clinicaltrials.gov

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