Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
ModernaTX, Inc. · NCT04159103
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Any age listed · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 1, Phase 2
- Target enrollment
- 77
- US sites recruiting
- 8
- Study start
- April 15, 2021
- Record last updated
- January 22, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the 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). 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).
Being studied: mRNA-3927
Conditions: Propionic Acidemia
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
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.
Recruiting US locations
Ronald Reagan UCLA Medical Center
Los Angeles, CA 90095
Site contact: Rosemary Silva-Garcia · rsilvagarcia@mednet.ucla.edu
Lucile Packard Children's Hospital Stanford
Stanford, CA 94304
Site contact: 650-497-0454 · hsrobin@stanford.edu
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, IL 60611
University of Michigan Hospitals
Ann Arbor, MI 48109
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, NY 10029
Duke University Medical System (Duke Health)
Durham, NC 27710
Site contact: Ming Xu · mingfen.xu@duke.edu
Children's Hospital of Philadelphia (CHOP)
Philadelphia, PA 19104
Site contact: Rebecca Madden · maddenr@chop.edu
Texas Children's Hospital
Houston, TX 77030
Site contact: Alyssa Tran · alyssat@bcm.edu
Study contacts (from the official record)
- Moderna WeCare Team · 1-866-663-3762 · WeCareClinicalTrials@modernatx.com
Similar studies in California
All nutrition & metabolism studies in California →- RecruitingHealthy volunteersPhase 1Overnight stay mentioned
A Study to Investigate the Relative Bioavailability and Safety of Different Oral Formulations of Elecoglipron in Healthy Participants
AstraZeneca
- Location
- Glendale, CA + 1 other US site
- Eligibility basics
- Ages 18–55
- RecruitingHealthy volunteersPhase 1/Phase 2
A Trial to Learn if ALN-PNP is Safe and Well Tolerated in Healthy Adults and Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Regeneron Pharmaceuticals
- Location
- Los Angeles, CA + 4 other US sites
- Eligibility basics
- Ages 18–75
- RecruitingHealthy volunteersPhase 1
The Effects of Dietary Supplementation on Serum Protein Glycosylation
University of California, Davis
- Location
- Sacramento, CA
- Eligibility basics
- Ages 18–45
- RecruitingHealthy volunteersPhase 1
TREAD: Time Restricted Eating Intervention for Alzheimer's Disease
University of California, San Diego
- Location
- San Diego, CA
- Eligibility basics
- Ages 60+