RecruitingPhase 1Phase 2NCT05130437

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

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

Sponsor

ModernaTX, Inc.

Enrollment

50 participants

Start Date

Nov 9, 2021

Study Type

INTERVENTIONAL

Conditions

Propionic Acidemia

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).

Eligibility

Min Age: 1 Year

Plain Language Summary

Simplified for easier understanding

This extension study evaluates the long-term safety of mRNA-3927, an investigational mRNA therapy being studied as a treatment for propionic acidemia (PA), a rare inherited metabolic disorder in which the body cannot properly break down certain proteins and fats. The study is open only to participants who previously completed the mRNA-3927-P101 trial, allowing them to continue receiving the same dose they were on before. Eligible participants must have completed the prior study's end-of-treatment visit, with the first dose in this extension study planned within 14 days of their last dose in the previous trial; those who had a liver or kidney transplant are not eligible. Participation involves receiving ongoing intravenous infusions of mRNA-3927 and being monitored for safety and any medical changes over time. This summary was prepared using AI to help patients understand the study in plain language.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALmRNA-3927

mRNA-3927 dispersion for IV infusion

Locations(22)

Ronald Reagan UCLA Medical Center

Los Angeles, California, United States

University of Stanford Medical Center

Palo Alto, California, United States

University of Michigan Hospitals

Ann Arbor, Michigan, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Duke University Medical System (Duke Health)

Durham, North Carolina, United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Texas Children's Hospital

Houston, Texas, United States

Hospital For Sick Children

Toronto, Ontario, Canada

AP-HM- Hôpital de La Timone

Marseille, France

Hôpital Necker - Enfants Malades

Paris, France

Fujita Health University Hospital

Toyoake-shi, Akita, Japan

Tohoku University Hospital

Sendai, Miyagi, Japan

Erasmus MC -Dr. Molewaterplein 40

Rotterdam, South Holland, Netherlands

Universitair Medisch Centrum Utrecht - PPDS

Utrecht, Netherlands

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, Saudi Arabia

King Abdullah Children's Specialist Hospital

Riyadh, Ar Riya, Saudi Arabia

Hospital Universitario Cruces

Barakaldo, Vizcaya, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Willink Biochemical Genetics Unit - Manchester

Manchester, England, United Kingdom

University Hospital Birmingham NHS Foundation Trust

Birmingham, West Midlands, United Kingdom

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, United Kingdom

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05130437

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