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.
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
120
CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.
Placebo is 0.9% normal saline solution and will be administered subcutaneously.
Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)
Herston, Queensland, Australia
Erasmus MC
Rotterdam, Netherlands
Adverse events
Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001
Time frame: Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Plasma PK
Plasma concentration of CMP-CPS-001
Time frame: 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
Time frame: 42 days (SAD) or 111 days (MAD, OTC) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
Ureagenesis rate test determination
Time frame: Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
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