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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
96
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
RECRUITINGAdverse events
Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001
Time frame: Screening (Day -36) until 42 days (SAD) or 112 days (MAD) after dosing
Plasma PK
Plasma concentration of CMP-CPS-001
Time frame: Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD) after dosing
Urinary excretion of CMP-CPS-001
Urine concentration of CMP-CPS-001
Time frame: 42 days (SAD) or 111 days (MAD) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
Ureagenesis rate test determination
Time frame: Run-in (Day -8) until 42 days (SAD) or 112 days (MAD) after dosing
Regulatory Affairs - Global Regulatory Clinical Services
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