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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05769621
Other study ID # mRNA-3927-P002
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date June 16, 2023
Est. completion date March 24, 2026

Study information

Verified date February 2024
Source ModernaTX, Inc.
Contact Moderna Clinical Trials Support Center
Phone 1-877-777-7187
Email clinicaltrials@modernatx.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


Description:

This study will abstract participants data from their medical records from approximately 2015-2025. All data will be abstracted retrospectively in 6-month increments.


Recruitment information / eligibility

Status Recruiting
Enrollment 100
Est. completion date March 24, 2026
Est. primary completion date March 24, 2026
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Abstraction Criteria: Participants' medical records are eligible for abstraction only if all of the following criteria apply: - Availability of evidence supportive of the participant having received a physician's diagnosis of PA. - Participant provided informed consent or assent, as applicable. - Participant has had at least one MDE in a consecutive 24-month period within the entire abstraction timeframe. Censoring Criteria: Participants medical records meeting the following censoring criterion will not be abstracted: - Participation in a clinical study of mRNA-3927 or another investigational agent.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
Australia Royal Children's Hospital Melbourne Melbourne Victoria
Canada Stollery Children's Hospital University of Alberta Edmonton Alberta
France CHU de Marseille - Hôpital de la Timone Marseille
France Hôpital Necker - Enfants Malades Paris Cedex 15 Paris
France CHU Toulouse Toulouse
France CHRU Nancy Hôpital des Enfants VandÅ“uvre-lès-Nancy Meurthe-et-Moselle
Italy Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN Firenze Toscana
Italy ASST di Monza - Azienda Ospedaliera San Gerardo Monza Monza A Brianza
Italy Azienda Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN Roma Lazio
Netherlands Erasmus MC Rotterdam
Spain Hospital Universitario Cruces Barakaldo
Spain Hospital Sant Joan de Deu Barcelona
Spain Hospital Universitario 12 de Octubre Madrid
Spain Hospital Universitario Raymon y Cajal Madrid
Spain Hospital Universitario Marques de Valdecilla Santander Cantabria
Spain Hospital Universitario Virgen del Rocio - PPDS Sevilla
Spain Hospital Universitari I Politecnic La Fe de Valencia Valencia
United Kingdom University Hospital Birmingham Birmingham
United Kingdom Great Ormond Street Hospital City of London London
United Kingdom Victoria Hospital Kirkcaldy Fife
United Kingdom St Mary's Hospital - PPDS Manchester Lancashire
United States University of Michigan Hospital Ann Arbor Michigan
United States Boston Children's Hospital Boston Massachusetts
United States Texas Children's Hospital Houston Texas
United States Johns Hopkins University Lutherville Maryland
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States University of Utah Health Care Salt Lake City Utah
United States Seattle Children's Hospital Seattle Washington
United States University of Stanford Medical Center Stanford California

Sponsors (1)

Lead Sponsor Collaborator
ModernaTX, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Annualized Metabolic Decompensation Events (MDEs) Up to 10 years
Secondary Annualized MDE Duration Up to 10 years
Secondary Number of Participants with MDE Symptoms MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy or increased seizure activity. Up to 10 years
Secondary Number of Annualized Hospitalizations Related to PA Up to 10 years
Secondary Annualized Duration of Hospitalizations Related to PA Up to 10 years
Secondary Number of Annualized Acute Healthcare Visits Related to PA Up to 10 years
Secondary Number of Participants who Received Medication During and Within ± 3 Days of an MDE Medications for standard long-term and acute management of PA, which may include, but are not limited to, anesthesia, antibiotics/antifungals, anticoagulation medications, antiepileptics, biotin supplementation, hydration treatment, PA treatment, pain medications, and transfusion related medications. Up to 10 years
Secondary Number of Participants Requiring Dietary Management Dietary management includes the following:
Prescribed protein intake during metabolic stability and during MDEs
Standard dietary management and sick-day diet
Up to 10 years
Secondary Change from Baseline in Biomarkers Up to 10 years
See also
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Recruiting NCT02890342 - Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
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Terminated NCT04836494 - A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia Phase 1
Completed NCT03484767 - "The MaP Study": Mapping the Patient Journey in MMA and PA
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Completed NCT00645879 - Anaplerotic Therapy in Propionic Acidemia Phase 1
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Terminated NCT05438485 - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
Completed NCT03159026 - Review of Charts From Amish/Mennonite Variant PA Patients
Recruiting NCT04159103 - Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia Phase 1/Phase 2