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

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age1+
SponsorModernaTX, Inc.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • mRNA-3927

    Biological/Vaccine

    mRNA-3927 dispersion for IV infusion

Treatment groups

50 Participants
are divided into 1 treatment group
Group A: mRNA-3927Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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 outcomes

1

Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)

Time frame
Baseline through End of Study Visit (up to 8 years)
2

Annualized Frequency of Investigator-reported MDE-related Hospitalizations

Time frame
Baseline through End of Study Visit (up to 8 years)
3

Annualized Frequency of Investigator-reported PA-related Hospitalizations

Time frame
Baseline through End of Study Visit (up to 8 years)
4

Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters

Time frame
Baseline through End of Study Visit (up to 8 years)

Other outcomes

Sponsors and contacts

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