Actively Recruiting

Phase 1
Phase 2
Age: 1Year +
All Genders
ID05130437

A Phase 1/2, Global, Open-Label Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Led by ModernaTX, Inc. · Updated on 2026-03-23

50

Participants Needed

22

Research Sites

104 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This research aims to evaluate the long-term safety of mRNA-3927 in people with propionic acidemia (PA) who previously took part in the mRNA-3927-P101 study. It is a phase 1/2, open-label extension study focusing on monitoring safety and clinical activity over an extended period. Participants will continue receiving mRNA-3927 at the same dose and schedule they had in the earlier study, with dose adjustments possible based on the sponsor's recommendation. The study includes two main periods: a treatment period where participants receive the study drug, followed by a 90-day follow-up period after the end of treatment. During the study, researchers will monitor participants for adverse events, serious side effects, and any events leading to stopping treatment. They will also track metabolic decompensation events and hospitalizations related to PA over up to eight years. Participants will have regular assessments to evaluate these outcomes and ensure ongoing safety throughout the study duration.

CONDITIONS

Brief Title

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Who Can Participate

Age: 1Year +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participated in Study mRNA-3927-P101
  • Completed the end-of-treatment or early termination visit in Study mRNA-3927-P101
  • Planned first dose in this study within 14 ±3 days of last dose in Study mRNA-3927-P101
Not Eligible

You will not qualify if you...

  • Not expected to benefit from continued mRNA-3927 treatment, according to the Investigator
  • Any medical or laboratory condition that may increase risk as judged by the Investigator
  • History of liver and/or kidney transplant

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 8 years

Participants receive mRNA-3927 at the dose and interval last received in the previous study. Dose adjustments may be made based on Sponsor recommendations.

Visits scheduled according to dosing and assessment needs during treatment

Follow-up

Duration - 90 days

Participants are monitored for safety and clinical activity for 90 days after completing treatment.

1 to 2 visits during follow-up

Trial Site Locations

Total: 22 locations

1

Ronald Reagan UCLA Medical Center

Los Angeles, California, United States, 90095

Not Yet Recruiting

2

University of Stanford Medical Center

Palo Alto, California, United States, 94304-1503

Actively Recruiting

3

University of Michigan Hospitals

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

4

Icahn School of Medicine at Mount Sinai

New York, New York, United States, 10029

Actively Recruiting

5

Duke University Medical System (Duke Health)

Durham, North Carolina, United States, 27710

Actively Recruiting

6

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

7

Texas Children's Hospital

Houston, Texas, United States, 77030

Actively Recruiting

8

Hospital For Sick Children

Toronto, Ontario, Canada, M5G 1X8

Actively Recruiting

9

AP-HM- Hôpital de La Timone

Marseille, France, 13005

Not Yet Recruiting

10

Hôpital Necker - Enfants Malades

Paris, France, 75019

Not Yet Recruiting

11

Fujita Health University Hospital

Toyoake-shi, Akita, Japan, 470-1192

Actively Recruiting

12

Tohoku University Hospital

Sendai, Miyagi, Japan, 980-8574

Actively Recruiting

13

Erasmus MC -Dr. Molewaterplein 40

Rotterdam, South Holland, Netherlands, 3015 GD

Not Yet Recruiting

14

Universitair Medisch Centrum Utrecht - PPDS

Utrecht, Netherlands, 3584 CX

Not Yet Recruiting

15

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, Saudi Arabia, 11211

Not Yet Recruiting

16

King Abdullah Children's Specialist Hospital

Riyadh, Ar Riya, Saudi Arabia, 14611

Not Yet Recruiting

17

Hospital Universitario Cruces

Barakaldo, Vizcaya, Spain, 48903

Not Yet Recruiting

18

Hospital Universitario 12 de Octubre

Madrid, Spain, 28041

Not Yet Recruiting

19

Willink Biochemical Genetics Unit - Manchester

Manchester, England, United Kingdom, M13 9WL

Actively Recruiting

20

University Hospital Birmingham NHS Foundation Trust

Birmingham, West Midlands, United Kingdom, B15 2TH

Actively Recruiting

21

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, United Kingdom, B4 6NH

Completed

22

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom, WC1N 3JH

Actively Recruiting

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Research Team

M

Moderna WeCare Team

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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