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Recruiting NCT05130437

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

Phase I / Phase II Interventional Propionic Acidemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: mRNA-3927.
Who it may be relevant to
Registry conditions: Propionic Acidemia. Basic parameters: from 1 year · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Canada, France, Japan, Netherlands +3
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Biological mRNA-3927
    mRNA-3927 dispersion for IV infusion

Primary outcome measures

  • Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation [Time frame: Baseline through End of Study Visit (up to 8 years)]
Secondary outcome measures (4)
  • Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs) [Time frame: Baseline through End of Study Visit (up to 8 years)]
  • Annualized Frequency of Investigator-reported MDE-related Hospitalizations [Time frame: Baseline through End of Study Visit (up to 8 years)]
  • Annualized Frequency of Investigator-reported PA-related Hospitalizations [Time frame: Baseline through End of Study Visit (up to 8 years)]
  • Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters [Time frame: Baseline through End of Study Visit (up to 8 years)]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 7 centers
  • Ronald Reagan UCLA Medical Center — Los Angeles
  • University of Stanford Medical Center — Palo Alto
  • University of Michigan Hospitals — Ann Arbor
  • Icahn School of Medicine at Mount Sinai — New York
  • Duke University Medical System (Duke Health) — Durham
  • The Children's Hospital of Philadelphia (CHOP) — Philadelphia
  • Texas Children's Hospital — Houston
United Kingdom · 4 centers
  • Willink Biochemical Genetics Unit - Manchester — Manchester
  • University Hospital Birmingham NHS Foundation Trust — Birmingham
  • Birmingham Women's and Children's NHS Foundation Trust — Birmingham
  • Great Ormond Street Hospital for Children NHS Foundation Trust — London
France · 2 centers
  • AP-HM- Hôpital de La Timone — Marseille
  • Hôpital Necker - Enfants Malades — Paris
Japan · 2 centers
  • Fujita Health University Hospital — Toyoake-shi
  • Tohoku University Hospital — Sendai
Netherlands · 2 centers
  • Erasmus MC -Dr. Molewaterplein 40 — Rotterdam
  • Universitair Medisch Centrum Utrecht - PPDS — Utrecht
Saudi Arabia · 2 centers
  • King Faisal Specialist Hospital & Research Centre — Riyadh
  • King Abdullah Children's Specialist Hospital — Riyadh
Spain · 2 centers
  • Hospital Universitario Cruces — Barakaldo
  • Hospital Universitario 12 de Octubre — Madrid
Canada · 1 center
  • Hospital For Sick Children — Toronto

Identifiers

NCT: NCT05130437 · mRNA-3927-P101-EXT · 2022-502911-12-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗