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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05769621




Registration number
NCT05769621
Ethics application status
Date submitted
3/03/2023
Date registered
15/03/2023
Date last updated
1/10/2024

Titles & IDs
Public title
A Retrospective Study to Characterize Participants With Propionic Acidemia and Methylmalonic Acidemia
Scientific title
A Retrospective Cohort Study to Characterize Propionic Acidemia Patients and Methylmalonic Acidemia
Secondary ID [1] 0 0
mRNA-3927-P002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Propionic Acidemia 0 0
Methylmalonic Acidemia 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Renal and Urogenital 0 0 0 0
Kidney disease

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
PA Participants - Participants with PA who meet all eligibility criteria for medical record abstraction.

MMA Participants - Participants with MMA who meet all eligibility criteria for medical record abstraction.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Annualized Metabolic Decompensation Events (MDEs)
Timepoint [1] 0 0
Up to 10 years
Secondary outcome [1] 0 0
Annualized MDE Duration
Timepoint [1] 0 0
Up to 10 years
Secondary outcome [2] 0 0
Number of Participants with MDE Symptoms
Timepoint [2] 0 0
Up to 10 years
Secondary outcome [3] 0 0
Number of Participants with MDE as Assessed by a Pre-Specified Severity Rating Scale
Timepoint [3] 0 0
Up to 10 years
Secondary outcome [4] 0 0
Number of Annualized Hospitalizations Related to PA or MMA
Timepoint [4] 0 0
Up to 10 years
Secondary outcome [5] 0 0
Annualized Duration of Hospitalizations Related to PA or MMA
Timepoint [5] 0 0
Up to 10 years
Secondary outcome [6] 0 0
Number of Annualized Urgent Healthcare Visits Related to PA or MMA
Timepoint [6] 0 0
Up to 10 years
Secondary outcome [7] 0 0
Number of Participants who Received Medication During and Within ± 3 Days of an MDE
Timepoint [7] 0 0
Up to 10 years
Secondary outcome [8] 0 0
Number of Participants Requiring Dietary Management
Timepoint [8] 0 0
Up to 10 years
Secondary outcome [9] 0 0
Change from Baseline in Biomarkers
Timepoint [9] 0 0
Up to 10 years

Eligibility
Key inclusion criteria
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 or MMA due to MUT deficiency as indicated by documented evidence.
* Participant provided informed consent (and assent, as applicable).
* Participant has had at least one MDE in 2015 or later before eligibility assessment.

Censoring Criteria:

Participants medical records meeting the following censoring criterion will not be abstracted:

* Participation in a clinical study of mRNA-3927, mRNA-3705, or another investigational agent.
* Other reasons of censorship of data include:
* Withdrawal of consent
* Death
* Lost to follow-up
* End of study period
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Retrospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
24/03/2026
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington
Country [7] 0 0
Canada
State/province [7] 0 0
Alberta
Country [8] 0 0
France
State/province [8] 0 0
Meurthe-et-Moselle
Country [9] 0 0
France
State/province [9] 0 0
Paris
Country [10] 0 0
France
State/province [10] 0 0
Marseille
Country [11] 0 0
France
State/province [11] 0 0
Toulouse
Country [12] 0 0
Italy
State/province [12] 0 0
Lazio
Country [13] 0 0
Italy
State/province [13] 0 0
Monza A Brianza
Country [14] 0 0
Italy
State/province [14] 0 0
Toscana
Country [15] 0 0
Netherlands
State/province [15] 0 0
Rotterdam
Country [16] 0 0
Spain
State/province [16] 0 0
Cantabria
Country [17] 0 0
Spain
State/province [17] 0 0
Barakaldo
Country [18] 0 0
Spain
State/province [18] 0 0
Barcelona
Country [19] 0 0
Spain
State/province [19] 0 0
Madrid
Country [20] 0 0
Spain
State/province [20] 0 0
Sevilla
Country [21] 0 0
Spain
State/province [21] 0 0
Valencia
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Fife
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Lancashire
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ModernaTX, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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) or Methylmalonic acidemia (MMA).
Trial website
https://clinicaltrials.gov/study/NCT05769621
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Moderna Clinical Trials Support Center
Address 0 0
Country 0 0
Phone 0 0
1-877-777-7187
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05769621