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CMP-CPS-001 · 1 trial · 2 indications
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...
CMP-CPS-001 is an investigational small molecule being developed by CAMP4 Therapeutics Corporation for OTC Deficiency, a rare genetic disorder. It is in Phase 1 clinical development and has received Rare Pediatric Disease and Orphan Drug designations from the FDA.
CMP-CPS-001 is being studied for OTC Deficiency, a rare inherited metabolic disorder. It is an investigational therapy and has not been approved for any indication. The Phase 1 trial includes healthy volunteers and participants with an abnormal heterozygous OTC genotype.
CMP-CPS-001 targets SYNGAP1, a gene. As a small molecule, it is designed to act on this gene target. SYNGAP1 is the molecular target specified for this investigational asset in its development for OTC Deficiency.
CMP-CPS-001 is being developed by CAMP4 Therapeutics Corporation, which trades under the ticker CAMP. The company is advancing the small molecule through Phase 1 clinical development for OTC Deficiency.
CMP-CPS-001 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 1 program includes one active trial that is currently not recruiting.
CMP-CPS-001 is being evaluated in the Phase 1 trial NCT06247670, titled Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype. The trial is active but not recruiting, with an enrollment of 120 participants across Australia and the Netherlands.