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

Recruiting now · Phase 1/Phase 2

Conditions studied: Propionic Acidemia

In brief

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).

Key facts

Study ID

NCT04159103

Run by

ModernaTX, Inc.

People needed

77

Starts

2021-04-15

Expected to finish

2027-08-31

Last updated by the study team

2026-01-22

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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 it is running

• Fujita Health University Hospital — Toyoake-shi, Aichi-ken, Japan (enrolling)
• Tohoku University Hospital — Sendai, Miyagi, Japan (enrolling)
• University of Michigan Hospitals — Ann Arbor, Michigan, United States (enrolling)
• Erasmus MC — Rotterdam, South Holland, Netherlands (enrolling)
• Icahn School of Medicine at Mount Sinai - Clinical Research Unit — New York, New York, United States (enrolling)
• Duke University Medical System (Duke Health) — Durham, North Carolina, United States (enrolling)
• Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Ronald Reagan UCLA Medical Center — Los Angeles, California, United States (enrolling)
• Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States (enrolling)
• Hospital Universitario Cruces — Barakaldo, Biscay, Spain (enrolling)
• Hospital Universitario 12 de Octubre — Madrid, Spain (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)
• University Hospital Birmingham NHS Foundation Trust — Birmingham, United Kingdom (enrolling)
• Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada (enrolling)
• Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (enrolling)
• Willink Biochemical Genetics Unit - PPDS — Manchester, United Kingdom (enrolling)
• Hospital For Sick Children — Toronto, Ontario, Canada (enrolling)
• Lucile Packard Children's Hospital Stanford — Stanford, California, United States (enrolling)
• Hôpital Necker - Enfants Malades — Paris, France (enrolling)
• CHU de Marseille - Hôpital de la Timone — Marseille, France
• Nicklaus Children's Hospital — Miami, Florida, United States
• University of South Florida - 12901 Bruce B Downs — Tampa, Florida, United States
• Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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