Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | PHASE1 | RECRUITING | 50 | — | — | Nov 9, 2021 | Dec 4, 2031 | Mar 23, 2026 | 22 | United States, Canada +6 |
| NCT04159103 | Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia | PHASE1 | RECRUITING | 77 | — | — | Apr 15, 2021 | Aug 31, 2027 | Jan 22, 2026 | 35 | United States, Canada +6 |
| Arm | Type | Description |
|---|---|---|
| mRNA-3927 | EXPERIMENTAL | Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation. |
| Part 1 (Dose Optimization), Part 2 (Pivotal Study), and Part 3 (Infants) | EXPERIMENTAL | 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 (Pivotal Study): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 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 26 doses or approximately 12 months. |
| Name | Type | Description |
|---|---|---|
| mRNA-3927 | BIOLOGICAL | mRNA-3927 dispersion for IV infusion |
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 e...