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NCT04159103 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Propionic Acidemia, sponsored by ModernaTX.

Recruiting
Registry status
Phase 1
Development phase
77
Enrollment target
20
Study locations

NCT04159103: Recruiting Phase 1 study of Propionic Acidemia, sponsored by ModernaTX.

NCT04159103 is a Phase 1 study of Propionic Acidemia that is actively recruiting participants, run by ModernaTX. The registered enrollment target is 77 participants, below the 213-participant average among 6 other Propionic Acidemia trials with a reported enrollment target (64% lower). The trial reports 20 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04159103, a Phase 1 study of Propionic Acidemia, is actively recruiting participants, sponsored by ModernaTX.

RECRUITING
Registry status
Phase 1
Development phase
77 participants
Enrollment target
20
Study locations

Study Summary

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).

Conditions Studied

Propionic Acidemia

Interventions

  • BIOLOGICAL mRNA-3927

Study Locations (20)

California

  • UCSD Altman Clinical and Transalational Research Institute Building - Los Angeles
  • Ronald Reagan UCLA Medical Center - Los Angeles
  • Lucile Packard Children's Hospital Stanford - Stanford

Florida

  • Nicklaus Children's Hospital - Miami
  • University of South Florida - 12901 Bruce B Downs - Tampa

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati
  • University Hospitals Cleveland Medical Center - 11100 Euclid Ave - Cleveland

Other

  • CHU de Marseille - Hôpital de la Timone - Marseille
  • Hôpital Necker - Enfants Malades - Paris

Illinois

  • Ann and Robert H Lurie Childrens Hospital of Chicago - Chicago

Maryland

  • Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit - Baltimore

Massachusetts

  • Boston Children's Hospital - Boston

Michigan

  • University of Michigan Hospitals - Ann Arbor

Trial Details

FieldValue
Enrollment Target 77 participants
Start Date 2021-04-15
Est. Completion 2027-08-31
Phase Phase 1
ModernaTX

90 total trials

What NCT04159103 shows while recruiting

NCT04159103 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 77 participants, a relatively small participant target, below the 213-participant average among 6 other Propionic Acidemia trials with a reported enrollment target (64% lower).

The record links to 1 condition, with Propionic Acidemia appearing as the primary indexed condition, and to 1 intervention - of which mRNA-3927 is the first listed.

NCT04159103 names 20 study sites across 15 states, led by California, Florida, Ohio.

Frequently Asked Questions

What is clinical trial NCT04159103 about?

NCT04159103 is a clinical study titled "Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia". 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 a...

What is the current status of trial NCT04159103?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 77 participants. The study started on 2021-04-15. Estimated completion is 2027-08-31.

What conditions does trial NCT04159103 study?

This clinical trial studies the following conditions: Propionic Acidemia.

What interventions are being tested in trial NCT04159103?

The interventions under investigation include: mRNA-3927 (BIOLOGICAL).

Who is sponsoring clinical trial NCT04159103?

This trial is sponsored by ModernaTX, which has 90 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04159103 being conducted?

This trial has 20 study locations across California, Florida, Illinois, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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More recruiting context for NCT04159103

Source: ClinicalTrials.gov NCT04159103, the US trial registry maintained by the National Library of Medicine. NCT04159103 (small enrollment · wide site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.