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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 designed to modulate gene expression as part of its mechanism of action in the treatment of rare diseases.
CMP-CPS-001 is being developed by CAMP4 Therapeutics Corporation, a biopharmaceutical company. The company is listed on the stock exchange 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. It 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 enrolling 120 participants and is active but not recruiting. The study is randomized, double-blind, and controlled, and is being conducted in Australia and the Netherlands.
CMP-CPS-001 is the drug name used in clinical development. No alternative names have been disclosed for this investigational compound.