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

NCT06247670 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: CPS-101
• Study Type: interventional
• Target: 120
• Locations: 2 countries
• Sites: 2

Brief 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.

Conditions

Healthy Volunteers; OTC Deficiency

Keywords

Healthy Volunteers; Antisense Oligonucleotides; Urea Cycle Disorder

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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

Study Arms (4)

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.

Drug: CMP-CPS-001; Other: Placebo

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.

Drug: CMP-CPS-001; Other: Placebo

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.

Drug: CMP-CPS-001; Other: 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.

Drug: CMP-CPS-001; Other: Placebo

Interventions

CMP-CPS-001 DRUG

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

Multiple Ascending Dose Part; OTC Heterozygous Participants in Australia; OTC Heterozygous Participants in EU; Single Ascending Dose Part

Placebo OTHER

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

Multiple Ascending Dose Part; OTC Heterozygous Participants in Australia; OTC Heterozygous Participants in EU; Single Ascending Dose Part

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• CAMP4 Therapeutics Corporation: lead

Study Sites (2)

Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)

Herston, Queensland, Australia

Location

Erasmus MC

Rotterdam, Netherlands

Location

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease; Urea Cycle Disorders, Inborn

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Gloria Wong, MD

  Q-Pharm Pty Ltd

  PRINCIPAL INVESTIGATOR

• Margreet Wagenmakers

  Erasmus Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2024
• First Posted: February 8, 2024
• Study Start: February 5, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); NL (1)