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
Sponsor study identifier
interventional
120
2 countries
2
Study Start
February 5, 2024
Actual (reported)Primary Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
January 22, 2024
Actual (reported)February 5, 2024
Actual (reported)February 8, 2024
Actual (reported)December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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
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
Adult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated.
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.
CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.
Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)
Herston, Queensland, Australia
Erasmus MC
Rotterdam, Netherlands
Gloria Wong, MD
Q-Pharm Pty Ltd
Margreet Wagenmakers
Erasmus Medical Center
First Submitted
January 22, 2024
First Posted
February 8, 2024
Study Start
February 5, 2024
Actual (reported)Primary Completion
December 2027
EstimatedAssessment of the Safety, Tolerability and Pharmacokinetics of AV078 in Healthy Volunteers
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.
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.
Placebo is 0.9% normal saline solution and will be administered subcutaneously.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
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Planned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
April 22, 2026
Record last verified: 2026-04
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