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CMP-CPS-001

Phase 1

Healthy Volunteers | Small molecule | Rare Disease |CAMP4 Therapeutics Corporation|Last Updated: Apr 22, 2026

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment120
FDA Designations
RARE_PEDIATRIC_DISEASEORPHAN_DRUG
Clinical trial landscape

CMP-CPS-001 · 1 trial · 2 indications

Phase 1 1
NCT06247670Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC GenotypeHealthy Volunteers
ACTIVE NOT_RECRUITING120 Analytics
PHASE1ACTIVE NOT_RECRUITING
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
Healthy Volunteers
NCT06247670120
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Study Endpoints
Primary Endpoints
Adverse events
Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing

Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001

Secondary Endpoints
Plasma PK
Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Urinary excretion of CMP-CPS-001
42 days (SAD) or 111 days (MAD, OTC) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Single Ascending Dose PartEXPERIMENTALAdult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated.
Multiple Ascending Dose PartEXPERIMENTALAdult 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 AustraliaEXPERIMENTALUp 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 EUEXPERIMENTALUp 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.
Interventions
NameTypeDescription
CMP-CPS-001DRUGCMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.
PlaceboOTHERPlacebo is 0.9% normal saline solution and will be administered subcutaneously.
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Eligibility Criteria
Age Range16 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites2

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...

Countries:AustraliaNetherlands
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT06247670Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT06247670studyFirstPostDate: changed