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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06247670 | Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype | PHASE1 | ACTIVE NOT_RECRUITING | 120 | — | — | Feb 5, 2024 | Dec 1, 2027 | Apr 22, 2026 | 2 | Australia, Netherlands |
Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001
| Arm | Type | Description |
|---|---|---|
| Single Ascending Dose Part | EXPERIMENTAL | Adult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated. |
| Multiple Ascending Dose Part | EXPERIMENTAL | Adult healthy volunteers in 4 cohorts of 12 will receive 3 monthly doses of either CMP-CPS-001 or placebo. Four dose levels will be evaluated. |
| OTC Heterozygous Participants in Australia | EXPERIMENTAL | Up to 12 clinically healthy female participants who have an abnormal heterozygous OTC genotype will receive 3 monthly doses of either CMP-CPS-001 or placebo. |
| OTC Heterozygous Participants in EU | EXPERIMENTAL | Up to 12 clinically healthy female participants who have an abnormal heterozygous OTC genotype will receive 3 monthly doses of either CMP-CPS-001 or placebo. |
| Name | Type | Description |
|---|---|---|
| CMP-CPS-001 | DRUG | CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously. |
| Placebo | OTHER | Placebo is 0.9% normal saline solution and will be administered subcutaneously. |
Inclusion Criteria: * Participants 18 (SAD, MAD, OTC in AUS) or 16 (OTC in EU) to 65 years inclusive at time of informed consent * BMI ≥18.0 and ≤32 kg/m2 at screening, and ≤110 kg * Willing and able to sign informed consent form * OTC cohorts: female and must have confirmed heterozygous OTC genoty...