Clinical trial NCT06247670: Study of CMP-CPS-001 in Healthy Volunteers
Official title: A Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Study of CMP-CPS-001 in Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Healthy Volunteers
Keywords: Healthy Volunteers
Antisense Oligonucleotides
Urea Cycle Disorder
Lead sponsor: CAMP4 Therapeutics Corporation
Sponsor class: INDUSTRY
First posted: 2024-02-08
Enrollment: 96
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: Inclusion Criteria:

* Healthy adults 18 to 55 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

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

Primary outcomes:
measure: Adverse events
timeFrame: Screening (Day -36) until 42 days (SAD) or 112 days (MAD) after dosing
Secondary outcomes:
measure: Plasma PK
timeFrame: Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD) after dosing
measure: Urinary excretion of CMP-CPS-001
timeFrame: 42 days (SAD) or 111 days (MAD) after dosing
measure: Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
timeFrame: Run-in (Day -8) until 42 days (SAD) or 112 days (MAD) after dosing
Other outcomes:
Locations:
city: Herston
country: Australia
facility: Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)
state: Queensland
Study officials:
affiliation: Q-Pharm Pty Ltd
name: Gloria Wong, MD
role: PRINCIPAL_INVESTIGATOR
References: