NCT04159103 · ModernaTX, Inc.
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
What this study is about
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).
View original scientific description
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).
Interventions
BIOLOGICAL
mRNA-3927
mRNA-3927 dispersion for IV infusion
Primary outcome measures
Part 1: Number of Participants with Treatment-emergent Adverse Event (TEAE), Serious Adverse Events (SAE) and TEAEs Leading to Discontinuation
Time frame: 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
Time frame: Pretreatment period (12 months before consent 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
Time frame: Day 1 up to Week 73
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 consent/assent if enrolled 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 screening 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 criteria 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 Investigator 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 criteria
- Participants of all ages are excluded from the study if during Screening 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 Investigator or Sponsor'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.
- Pregnant or breastfeeding.
- 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.
Where
- Los Angeles, California
- Stanford, California
- Miami, Florida
- Tampa, Florida
- Chicago, Illinois
- Baltimore, Maryland
- Boston, Massachusetts
- Ann Arbor, Michigan
- New York, New York
- Durham, North Carolina
- Cincinnati, Ohio
- Cleveland, Ohio
And 2 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jan 22, 2026 · Source of record for eligibility and locations