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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Healthy Volunteers; OTC Deficiency
• Intervention / Treatment: Drug: CMP-CPS-001; Other: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 120
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Herston, Queensland, Australia
      • Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)
• Netherlands
  • Rotterdam, Netherlands
    • Erasmus MC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single Ascending Dose Part; Adult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated.	Drug: CMP-CPS-001; CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.; Other: Placebo; Placebo is 0.9% normal saline solution and will be administered subcutaneously.
Experimental: Multiple Ascending Dose Part; 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.	Drug: CMP-CPS-001; CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.; Other: Placebo; Placebo is 0.9% normal saline solution and will be administered subcutaneously.
Experimental: OTC Heterozygous Participants in Australia; 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.	Drug: CMP-CPS-001; CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.; Other: Placebo; Placebo is 0.9% normal saline solution and will be administered subcutaneously.
Experimental: OTC Heterozygous Participants in EU; 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.	Drug: CMP-CPS-001; CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.; Other: Placebo; Placebo is 0.9% normal saline solution and will be administered subcutaneously.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events	Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001	Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma PK	Plasma concentration of CMP-CPS-001	Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Urinary excretion of CMP-CPS-001	Urine concentration of CMP-CPS-001	42 days (SAD) or 111 days (MAD, OTC) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis	Ureagenesis rate test determination	Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing

Collaborators and Investigators

• Sponsor: CAMP4 Therapeutics Corporation
• Investigators: Principal Investigator: Gloria Wong, MD, Q-Pharm Pty Ltd; Principal Investigator: Margreet Wagenmakers, Erasmus Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): February 5, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: January 22, 2024
• First Submitted That Met QC Criteria: January 30, 2024
• First Posted (Actual): February 8, 2024

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 17, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Healthy Volunteers; Urea Cycle Disorder; Antisense Oligonucleotides
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Genetic Diseases, X-Linked; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Amino Acid Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Ornithine Carbamoyltransferase Deficiency Disease; Urea Cycle Disorders, Inborn
• Other Study ID Numbers: CPS-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes