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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
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Scientific title
A Retrospective Cohort Study to Characterize Propionic Acidemia Patients and Methylmalonic Acidemia
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Secondary ID [1]
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mRNA-3927-P002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Propionic Acidemia
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Methylmalonic Acidemia
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Condition category
Condition code
Metabolic and Endocrine
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Metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Respiratory
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Other respiratory disorders / diseases
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Renal and Urogenital
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Kidney disease
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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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.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Annualized Metabolic Decompensation Events (MDEs)
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Assessment method [1]
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Timepoint [1]
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Up to 10 years
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Secondary outcome [1]
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Annualized MDE Duration
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Assessment method [1]
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Timepoint [1]
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Up to 10 years
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Secondary outcome [2]
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Number of Participants with MDE Symptoms
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Assessment method [2]
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MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy, change in behavior or level of consciousness or increased seizure activity.
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Timepoint [2]
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Up to 10 years
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Secondary outcome [3]
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Number of Participants with MDE as Assessed by a Pre-Specified Severity Rating Scale
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Assessment method [3]
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Timepoint [3]
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Up to 10 years
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Secondary outcome [4]
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Number of Annualized Hospitalizations Related to PA or MMA
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Assessment method [4]
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Timepoint [4]
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Up to 10 years
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Secondary outcome [5]
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Annualized Duration of Hospitalizations Related to PA or MMA
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Assessment method [5]
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Timepoint [5]
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Up to 10 years
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Secondary outcome [6]
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Number of Annualized Urgent Healthcare Visits Related to PA or MMA
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Assessment method [6]
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Timepoint [6]
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Up to 10 years
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Secondary outcome [7]
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Number of Participants who Received Medication During and Within ± 3 Days of an MDE
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Assessment method [7]
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Medications for standard long-term and acute management of PA or MMA, which may include, but are not limited to, anesthesia, antibiotics/antifungals, anticoagulation medications, antiepileptics, biotin or cobalamin supplementation, hydration treatment, PA treatment, MMA treatment, pain medications, and transfusion related medications.
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Timepoint [7]
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Up to 10 years
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Secondary outcome [8]
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Number of Participants Requiring Dietary Management
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Assessment method [8]
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Dietary management includes the following: * Prescribed protein intake during metabolic stability and during MDEs * Sick-day diet as available.
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Timepoint [8]
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Up to 10 years
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Secondary outcome [9]
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Change from Baseline in Biomarkers
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Assessment method [9]
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Timepoint [9]
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Up to 10 years
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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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose
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Duration
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Selection
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Timing
Retrospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/06/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
24/03/2026
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Actual
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Sample size
Target
200
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Royal Children's Hospital Melbourne - Melbourne
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Recruitment postcode(s) [1]
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3052 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Maryland
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United States of America
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Michigan
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United States of America
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Pennsylvania
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Country [5]
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United States of America
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Texas
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Country [6]
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United States of America
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Washington
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Country [7]
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Canada
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State/province [7]
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Alberta
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Country [8]
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France
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State/province [8]
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Meurthe-et-Moselle
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Country [9]
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France
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State/province [9]
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Paris
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Country [10]
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France
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State/province [10]
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Marseille
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France
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State/province [11]
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Toulouse
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Italy
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Lazio
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Italy
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State/province [13]
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Monza A Brianza
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Country [14]
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Italy
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State/province [14]
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Toscana
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Country [15]
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Netherlands
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State/province [15]
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Rotterdam
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Country [16]
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Spain
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State/province [16]
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Cantabria
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Country [17]
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Spain
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State/province [17]
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Barakaldo
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Country [18]
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Spain
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State/province [18]
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Barcelona
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Spain
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Madrid
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Country [20]
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Spain
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State/province [20]
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Sevilla
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Spain
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State/province [21]
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Valencia
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Country [22]
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United Kingdom
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State/province [22]
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Fife
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Country [23]
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United Kingdom
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State/province [23]
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Lancashire
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Country [24]
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United Kingdom
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State/province [24]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
ModernaTX, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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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).
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Trial website
https://clinicaltrials.gov/study/NCT05769621
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Moderna Clinical Trials Support Center
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Address
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Country
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Phone
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1-877-777-7187
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Fax
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Email
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[email protected]
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05769621
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