mRNA-3927 for Propionic Acidemia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Propionic AcidemiamRNA-3927 - Biological
Eligibility
Any Age
All Sexes
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Study Summary

This trial will test the long-term safety of a medication for propionic acidemia, a rare metabolic disorder.

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

1 Primary · 10 Secondary · Reporting Duration: Baseline up to 8 years

Year 8
Number of Clinically Significant Events
Number of Days Participants Missed School and Workdays
Number of Healthcare Resource Utilization Visits
Number of Metabolic Decompensation Events (MDEs)
Year 8
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Year 8
Change From Baseline in Pediatric Quality-of-Life Inventory (PedsQL)
Change From Baseline in Plasma 2-Methylcitrate (2-MC) Levels
Change From Baseline in Plasma 3-Hydroxypropionic Acid (3-HP) Levels
Year 8
Number of Anti-PCC Antibodies
Number of Anti-Polyethylene Glycol Antibodies
Year 8
Pharmacokinetic (PK): Propionyl-CoA Carboxylase (PCC) Subunit α (PCCA) and Propionyl-CoA Carboxylase Subunit β (PCCB) mRNA (Serum) and SM-86 (Plasma) Levels

Trial Safety

Safety Progress

1 of 3

Similar Trials

Trial Design

1 Treatment Group

mRNA-3927
1 of 1

Experimental Treatment

36 Total Participants · 1 Treatment Group

Primary Treatment: mRNA-3927 · No Placebo Group · Phase 1 & 2

mRNA-3927
Biological
Experimental Group · 1 Intervention: mRNA-3927 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline up to 8 years

Who is running the clinical trial?

ModernaTX, Inc.Lead Sponsor
68 Previous Clinical Trials
50,290,883 Total Patients Enrolled
2 Trials studying Propionic Acidemia
139 Patients Enrolled for Propionic Acidemia

Eligibility Criteria

Age Any Age · All Participants · 2 Total Inclusion Criteria

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