Testing mRNA-3927 for propionic acidemia
Official title: Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia
- Phase 1
- 1 group
- Sites in Edmonton and Toronto
- Recruiting
Interventions
- Biological therapy
mRNA-3927
mRNA-3927 dispersion for IV infusion
Canadian Sites (2)
2 of 2 recruiting
- Recruiting
Stollery Children's Hospital University of Alberta
Edmonton, Alberta
- Recruiting
Hospital For Sick Children
Toronto, Ontario
Eligibility Criteria
See who this study is looking for24 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
- +≥ 8 years of age at the time of consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →/assent if enrolledEnrolmentThe number of participants a study plans to include, or has included.Read more → as 1 of the first 2 participants in Part 1.
- +≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1.
- +Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations).
- +Part 2 only: At least one documented MDE in the 12-month period before consent.
- +Participants \<1 Year of Age :
- +Identification by newborn screeningScreeningThe checks done before joining, to see whether a study fits.Read more → shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteriaEligibility criteriaThe full list of requirements for taking part in a study.Read more → are met but would not be enrolled until diagnosis of PA is confirmed.
- +For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → may interfere with the interpretation of study results.
- +Body weight ≥3 kilograms (kg) at Screening.
- +At least 1 documented PA-related event prior to Screening defined as the following criteria:
- +Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR
- +Meeting the criteria of MDE definition, OR
- +Evidence of laboratory abnormalities as evidenced by at least one of the following:
- +Metabolic acidosis with elevated anion gap.
- +Acute hyperammonemia.
- +Neutropenia or thrombocytopenia.
Exclusion
- −Participants of all ages are excluded from the study if during ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more → any of the following criteria apply:
- −Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → or SponsorSponsorThe organisation responsible for the study overall.Read more →'s opinion that could interfere with or limit the participation in the study.
- −Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis.
- −History of organ transplantation or planned organ transplantation during the period of study participation.
- −Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction.
- −Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
- −Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.
- −Pregnant or breastfeeding.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
Everyone in this study is in one group and receives mRNA-3927.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
There is only one group in this study, so there is no assignment to different treatments.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 77 people.
The study is currently expected to finish around August 2027.
The main measurement is taken over: Day 1 (initial mRNA-3927 dose) up to Week 150 (End of Study).
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Part 1: Number of Participants with Treatment-emergent Adverse EventAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAE), Serious Adverse EventsSerious adverse eventAn adverse event meeting a formal severity threshold, such as requiring hospital admission.Read more → (SAE) and TEAEs Leading to Discontinuation — measured over Day 1 (initial mRNA-3927 dose) up to Week 150 (End of Study).
Part 2: Change in Annualized Frequency of Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) During 12-month Treatment Period With mRNA-3927 Compared to Annualized Frequency of CEC-adjudicated MDE During Pretreatment Period — measured over Pretreatment period (12 months before consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → to first mRNA-3927 dose in the study) up to Month 12.
Part 3: Number of Participants with TEAEs, SAEs, Adverse Events (AEs) of Special Interest (AESIs) and TEAEs Leading to Discontinuation — measured over Day 1 up to Week 73.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT04159103 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About mRNA-3927Biological
mRNA-3927 dispersion for IV infusion
From the trial registry — its own words, unedited.
What a biological is here: A treatment made from or by living systems — such as antibodies, vaccines, or cell-based therapies.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
There is only one group in this study, so there is no assignment to different treatments.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Day 1 (initial mRNA-3927 dose) up to Week 150 (End of Study).
The study as a whole is currently expected to finish around 2027-08-31.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 77 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 4 locations, including: Edmonton, Alberta, Canada; Toronto, Ontario, Canada; Ann Arbor, Michigan, United States; New York, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).
Other Sites (2)
University of Michigan Hospitals
Ann Arbor, Michigan, United States
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, New York, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.