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RNA Therapy

mRNA-3927 for Propionic Acidemia

Q: Is enrollment still available for this investigation?

Indeed, clinicaltrials.gov declares that this research is still recruiting participants. It was originally posted on April 15th 2021 and has since been amended as of October 28th 2022. The study seeks to acquire 36 volunteers from 10 distinct medical centres.

Q: How many centers are engaged in this research endeavor?

For this clinical trial, there are a total of 10 medical sites including the David Geffen School of Medicine UCLA (Los Angeles), Children's Hospital of Philadelphia (Philadelphia), and Cincinnati Children's Hospital (Cincinnati). Additionally, 7 other locations also offer this treatment.

Q: What is the ceiling of participants enrolled in this research endeavour?

Affirmative. Clinicaltrials.gov data reveals that the recruitment phase of this trial, which began on April 15th 2021 is ongoing. 36 individuals are needed to fill slots at 10 medical institutions across the country.

Phase 1 & 2

Recruiting

Research Sponsored by ModernaTX, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (PCCA and/or PCCB mutations)

Participant must be ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 (initial mrna-3927 dose) up to week 150 (end of study)

Awards & highlights

Study Summary

This trial will test a new treatment for propionic acidemia, a rare genetic disorder. The study is designed to assess the safety, pharmacokinetics, and pharmacodynamics of different doses of the treatment.

Who is the study for?

This trial is for people aged 1 year and older with a genetic confirmation of propionic acidemia (PA). Participants must have had at least one metabolic decompensation event in the past year. They can't join if they have severe heart failure, organ transplants, certain heart rhythm issues, recent COVID-19 vaccination, or poor kidney function.Check my eligibility

What is being tested?

The study tests mRNA-3927's safety and effectiveness in treating PA. It has two parts: first to find a safe dose that works (Dose Optimization), then to further assess this dose's efficacy and safety (Dose Expansion) by monitoring biomarkers.See study design

What are the potential side effects?

Potential side effects are not specified but will be monitored throughout the trial as part of assessing mRNA-3927’s safety profile. This includes any adverse reactions participants might experience during the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with PA through genetic testing.

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I am at least 1 year old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 (initial mrna-3927 dose) up to week 150 (end of study)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 (initial mrna-3927 dose) up to week 150 (end of study)

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 (initial mrna-3927 dose) up to week 150 (end of study) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part 1: Number of Participants with an Adverse Event (AE), Serious AE (SAE) and AE Leading to Discontinuation

Part 2: Number of Metabolic Decompensation Events (MDEs)

Part 3: Number of Participants with Treatment- emergent Adverse Event (TEAEs), SAEs, AEs of Special Interest and TEAEs Leading to Discontinuation

Secondary outcome measures

Part 1: Area Under the Effect Versus Time Curve (AUEC) of 2-MC and 3-HP after Single and Repeated Administrations of mRNA-3927

Part 1: Area Under the Plasma Concentration-Time Curve (AUC) of PCCA and PCCB mRNAs

Part 1: Change From Baseline in Plasma 2-Methylcitrate (2-MC) and 3-Hydroxypropionic Acid (3-HP) Levels After Single and Repeated Administrations of mRNA-3927

+13 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Part 1 (Dose Optimization), Part 2 (Dose Expansion), and Part 3 (Infants)Experimental Treatment1 Intervention

Part 1 (Dose Optimization): Participants (≥1 year of age) will receive single dose of mRNA-3927 by intravenous (IV) infusion every 2 weeks (Q2W) or every 3 weeks (Q3W) for up to 10 doses. Part 2 (Dose Expansion): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 12 months. Part 3: Participants (<1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 12 months.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

ModernaTX, Inc.Lead Sponsor

108 Previous Clinical Trials

61,379,249 Total Patients Enrolled

3 Trials studying Propionic Acidemia

233 Patients Enrolled for Propionic Acidemia

Media Library

mRNA-3927 (RNA Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04159103 — Phase 1 & 2

Propionic Acidemia Research Study Groups: Part 1 (Dose Optimization), Part 2 (Dose Expansion), and Part 3 (Infants)

Propionic Acidemia Clinical Trial 2023: mRNA-3927 Highlights & Side Effects. Trial Name: NCT04159103 — Phase 1 & 2

mRNA-3927 (RNA Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04159103 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is enrollment still available for this investigation?

"Indeed, clinicaltrials.gov declares that this research is still recruiting participants. It was originally posted on April 15th 2021 and has since been amended as of October 28th 2022. The study seeks to acquire 36 volunteers from 10 distinct medical centres."

Answered by AI

How many centers are engaged in this research endeavor?

"For this clinical trial, there are a total of 10 medical sites including the David Geffen School of Medicine UCLA (Los Angeles), Children's Hospital of Philadelphia (Philadelphia), and Cincinnati Children's Hospital (Cincinnati). Additionally, 7 other locations also offer this treatment."

Answered by AI

What is the ceiling of participants enrolled in this research endeavour?

"Affirmative. Clinicaltrials.gov data reveals that the recruitment phase of this trial, which began on April 15th 2021 is ongoing. 36 individuals are needed to fill slots at 10 medical institutions across the country."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

2021. "Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04159103.