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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 studied in healthy volunteers and in participants with ornithine transcarbamylase (OTC) deficiency, a rare disease. It is currently in Phase 1 clinical development and has not been approved by the FDA.
CMP-CPS-001 targets the SYNGAP1 gene. It is being developed as a small molecule therapeutic for rare disease indications, including OTC deficiency.
CMP-CPS-001 is being developed by CAMP4 Therapeutics Corporation, a biopharmaceutical company. The company's stock trades under the ticker symbol CAMP.
CMP-CPS-001 is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The drug has been granted orphan drug designation and rare pediatric disease designation by the FDA.
CMP-CPS-001 is being studied in a Phase 1 clinical trial with the identifier NCT06247670. This trial is active but not recruiting and has an enrollment of 120 participants. The study is being conducted in Australia and the Netherlands.
CMP-CPS-001 is the primary name for this investigational drug. No alternative names have been established for this asset.