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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06488313 | A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD | PHASE2 | RECRUITING | 9 | — | — | Nov 4, 2024 | Sep 1, 2026 | Mar 17, 2025 | 1 | United States |
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
| Arm | Type | Description |
|---|---|---|
| ARCT-810 | EXPERIMENTAL | Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals. |
| Name | Type | Description |
|---|---|---|
| ARCT-810 | BIOLOGICAL | ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP). |
Inclusion Criteria: 1. Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent. 2. Males and Females aged ≥12 years, at Screening. 3. Documented clinical diagnosis of OTC deficiency. 4. History of symptomatic hyperammonemia or elevat...