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RecruitingPhase 1/Phase 2

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

ModernaTX, Inc. · NCT04159103

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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
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia (NCT04159103) | Bored Studies