For Healthcare Professionals

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

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About the study

This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics (PK), and pharmacodynamics (PD) of different doses of mRNA-3927 in participants affected by PA as part of the Dose Optimization phase.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. Participant must be ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants.
  2. Participant must be ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants.
  3. Confirmed diagnosis of propionic acidemia based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunit α [PCCA] and/or propionyl CoA carboxylase subunit β [PCCB] mutations)

EXCLUSION CRITERIA

  1. Estimated glomerular filtration rate <30 milliliters (mL)/minute/1.73 square meter (m^2) as estimated by Schwartz formula for participants < 18 years of age or the Chronic Kidney Disease Epidemiology Collaboration creatinine based formula for participants ≥ 18 years of age or for participants of all ages receiving chronic dialysis.
  2. History of organ transplantation or planned organ transplantation during the period of study participation.
  3. Corrected QT interval (QTc) >480 milliseconds (ms) using Bazett's correction.
  4. Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
  5. COVID-19 vaccination (generally 2 doses or a booster) within 6 weeks between their last COVID-19 vaccination dose and first study drug administration.
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall 1-877-777-7187Email iconEmail Study Center

Study’s details


Contition

Propionic Acidemia

Age

1+

Phase

Phase 1/Phase 2

Participants needed

36

Est. Completion Date

Jan 2027

Treatment type

Interventional


Sponsor

ModernaTX, Inc.

ClinicalTrials.gov identifier

NCT04159103

Study number

mRNA-3927-P101

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