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NCT05130437PHASE1, PHASE2Recruiting

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

ModernaTX, Inc.

Start Date

11/9/2021

Completion Date

12/4/2031

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).

Detailed Description

The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met. The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.

Eligibility Criteria

Age Range: 1 years to No maximum

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Interventions

BIOLOGICAL

mRNA-3927

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Conditions

Propionic Acidemia

Locations

Ronald Reagan UCLA Medical Center

Los Angeles, California 90095

United States

University of Stanford Medical Center

Palo Alto, California 94304-1503

United States

University of Michigan Hospitals

Ann Arbor, Michigan 48109

United States

Icahn School of Medicine at Mount Sinai

New York, New York 10029

United States

Duke University Medical System (Duke Health)

Durham, North Carolina 27710

United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania 19104

United States

Texas Children's Hospital

Houston, Texas 77030

United States

Hospital For Sick Children

Toronto, Ontario M5G 1X8

Canada

AP-HM- Hôpital de La Timone

Marseille, 13005

France

Hôpital Necker - Enfants Malades

Paris, 75019

France

Fujita Health University Hospital

Toyoake-shi, Akita 470-1192

Japan

Tohoku University Hospital

Sendai, Miyagi 980-8574

Japan

Erasmus MC -Dr. Molewaterplein 40

Rotterdam, South Holland 3015 GD

Netherlands

Universitair Medisch Centrum Utrecht - PPDS

Utrecht, 3584 CX

Netherlands

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya 11211

Saudi Arabia

King Abdullah Children's Specialist Hospital

Riyadh, Ar Riya 14611

Saudi Arabia

Hospital Universitario Cruces

Barakaldo, Vizcaya 48903

Spain

Hospital Universitario 12 de Octubre

Madrid, 28041

Spain

Willink Biochemical Genetics Unit - Manchester

Manchester, England M13 9WL

United Kingdom

University Hospital Birmingham NHS Foundation Trust

Birmingham, West Midlands B15 2TH

United Kingdom

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands B4 6NH

United Kingdom

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH

United Kingdom