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

NCT05130437 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: mRNA-3927-P101-EXT2022-502911-12-00
• Study Type: interventional
• Target: 50
• Locations: 8 countries
• Sites: 22

Brief Summary

The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).

Conditions

Propionic Acidemia

Keywords

mRNA-3927; Propionic Aciduria; Metabolism, Inborn Errors; Genetic Diseases; Inborn Amino Acid Metabolism, Inborn Errors; Acidosis; Acid-Base Imbalance; Metabolic Diseases; Organic Acidemias; Moderna; mRNA

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation

  Baseline through End of Study Visit (up to 8 years)

Secondary Outcomes (4)

• Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)

  Baseline through End of Study Visit (up to 8 years)

• Annualized Frequency of Investigator-reported MDE-related Hospitalizations

  Baseline through End of Study Visit (up to 8 years)

• Annualized Frequency of Investigator-reported PA-related Hospitalizations

  Baseline through End of Study Visit (up to 8 years)

• Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters

  Baseline through End of Study Visit (up to 8 years)

Study Arms (1)

mRNA-3927

EXPERIMENTAL

Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.

Biological: mRNA-3927

Interventions

mRNA-3927 BIOLOGICAL

mRNA-3927 dispersion for IV infusion

mRNA-3927

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participated in Study mRNA-3927-P101.
• Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.

You may not qualify if:

• Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
• Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
• History of liver and/or kidney transplant.

Sponsors & Collaborators

• ModernaTX, Inc.: lead

Study Sites (22)

Ronald Reagan UCLA Medical Center

Los Angeles, California, 90095, United States

NOT YET RECRUITING

University of Stanford Medical Center

Palo Alto, California, 94304-1503, United States

RECRUITING

University of Michigan Hospitals

Ann Arbor, Michigan, 48109, United States

RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

Duke University Medical System (Duke Health)

Durham, North Carolina, 27710, United States

RECRUITING

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

AP-HM- Hôpital de La Timone

Marseille, 13005, France

NOT YET RECRUITING

Hôpital Necker - Enfants Malades

Paris, 75019, France

NOT YET RECRUITING

Fujita Health University Hospital

Toyoake-shi, Akita, 470-1192, Japan

RECRUITING

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

RECRUITING

Erasmus MC -Dr. Molewaterplein 40

Rotterdam, South Holland, 3015 GD, Netherlands

NOT YET RECRUITING

Universitair Medisch Centrum Utrecht - PPDS

Utrecht, 3584 CX, Netherlands

NOT YET RECRUITING

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, 11211, Saudi Arabia

NOT YET RECRUITING

King Abdullah Children's Specialist Hospital

Riyadh, Ar Riya, 14611, Saudi Arabia

NOT YET RECRUITING

Hospital Universitario Cruces

Barakaldo, Vizcaya, 48903, Spain

NOT YET RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

NOT YET RECRUITING

Willink Biochemical Genetics Unit - Manchester

Manchester, England, M13 9WL, United Kingdom

RECRUITING

University Hospital Birmingham NHS Foundation Trust

Birmingham, West Midlands, B15 2TH, United Kingdom

RECRUITING

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, B4 6NH, United Kingdom

COMPLETED

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Propionic Acidemia; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Amino Acid Metabolism, Inborn Errors; Acidosis; Acid-Base Imbalance; Metabolic Diseases

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases

Central Study Contacts

Moderna WeCare Team

CONTACT

1-866-663-3762; WeCareClinicalTrials@modernatx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2021
• First Posted: November 23, 2021
• Study Start: November 9, 2021
• Primary Completion (Estimated): December 4, 2029
• Study Completion (Estimated): December 4, 2031
• Last Updated: March 23, 2026

Record last verified: 2026-03

Locations

JP (2); CA (1); GB (4); US (7); NL (2); ES (2); FR (2); SA (2)