Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06247670

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Healthy Volunteers Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases OTC Deficiency Ornithine Carbamoyltransferase Deficiency Disease Urea Cycle Disorders, Inborn

Age range

16 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd), Herston, Queensland, Australia

Loading trial locations.

About this study

This is a randomized, double-blind (Sponsor-open), and placebo-controlled study.

The SAD part will be conducted in approximately 48 healthy volunteers, in 4 cohorts of 12, randomized 3:1 to receive a single subcutaneous dose of CMP-CPS-001 or placebo. Participants will be followed for 42 days after dosing.

The MAD part will be conducted in approximately 48 healthy volunteers, in 4 cohorts of 12, randomized 3:1 to receive 3 monthly doses of CMP-CPS-001 or placebo. Participants will be followed for 56 days after the last dose.

Up to 12 participants in Australia and up to 12 participants in EU with abnormal heterozygous OTC genotype will be randomized 3:1 to receive 3 monthly doses of CMP-CPS-001 or placebo. Participants will be followed for 56 days after the last dose.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

  1. Adverse events

    Time frame: 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 outcomes

  1. Plasma PK

    Time frame: Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD, OTC) after dosing

    Plasma concentration of CMP-CPS-001

  2. Urinary excretion of CMP-CPS-001

    Time frame: 42 days (SAD) or 111 days (MAD, OTC) after dosing

    Urine concentration of CMP-CPS-001

  3. Pharmacodynamic effect of CMP-CPS-001 on ureagenesis

    Time frame: Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing

    Ureagenesis rate test determination

Sponsors and collaborators

Lead sponsor

CAMP4 Therapeutics Corporation

Industry

Registry information

Official study title

A Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Phase 1 Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous Ornithine Transcarbamylase (OTC) Genotype

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Feb 8, 2024
Registry last updated
Apr 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.