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

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2043250110

Enrollment

Registered

2025-10-07

Start date

2025-04-18

Completion date

Unknown

Last updated

2025-10-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Propionic Acidemia

Interventions

Patients enrolled in this long-term extension study will receive treatment using the same dosing regimen as in the mRNA-3927-P101 study (dose: 0.45 - 0.9 mg/kg), administered every 2 weeks, with a min

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -Participated in Study mRNA-3927-P101. -Completed the EOT Visit in Study mRNA-3927-P101 within 10 days of first dose of mRNA-3927 in current study.

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
Evaluate the long-term efficacy of mRNA-3927 as determined by the frequency of MDEs.	—

Countries

Canada, France, Italy, Japan, Netherlands, Saudi Arabia, Spain, U.K., U.S.A.

Contacts

Public ContactMasashi Kinoshita

PPD-SNBL K.K.

masashi.kinoshita@thermofisher.com+81-80-2707-5974

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Feb 4, 2026