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Active, not recruiting Phase 1

Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype

NCT06247670 · tracked via the Priya Life Science Australia tracker
Sponsor
CAMP4 Therapeutics Corporation
Phase
Phase 1
Started
2024-02-05
Last updated
2026-04-22

Condition(s) studied

Healthy VolunteersOTC Deficiency

Investigational drug(s) / intervention(s)

CMP-CPS-001Placebo

CMP-CPS-001: CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.

Placebo: Placebo is 0.9% normal saline solution and will be administered subcutaneously.

Study summary

The objective of this clinical study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of CMP-CPS-001 administered as a subcutaneous injection in adult healthy volunteers and participants with abnormal heterozygous OTC genotype.

Eligibility

Sex
ALL
Min age
16 Years
Max age
65 Years
Healthy volunteers
Accepted
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 genotype Exclusion Criteria: * Any significant disease or disorder which, in the opinion of the Investigator, may either put the study participant at risk because of participation in the study, may influence the results of the study, or may affect the study participant's ability to participate in the study * Clinically relevant illness within 7 days before the first dose of study drug * History of intolerance to subcutaneous injection or relevant abdominal scarring * Laboratory results outside normal ranges at screening and judged as clinically relevant by the Investigator for liver function, kidney function, and platelets * Positive viral serology test results for human immunodeficiency virus type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus antibody * Any other safety laboratory result considered clinically significant and unacceptable by the Investigator

Primary outcome measure(s)

  • 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

Trial sites (2)

FacilityCityRegionStatus
Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd) Herston Queensland
Erasmus MC Rotterdam Netherlands
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06247670 on ClinicalTrials.gov ↗ ← All trials in Australia