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

CAMP4 Therapeutics

Status and phase: Active, not recruiting; Phase 1

Conditions: Healthy Volunteers; OTC Deficiency

Treatments: Other: Placebo; Drug: CMP-CPS-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT06247670

CPS-101

Details and patient eligibility

About

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.

Full 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.

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.

Enrollment: 120 estimated patients

Sex: All

Ages: 16 to 65 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• 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

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

120 participants in 4 patient groups

Single Ascending Dose Part

Experimental group

Description:

Adult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated.

Treatment:

Drug: CMP-CPS-001

Other: Placebo

Multiple Ascending Dose Part

Experimental group

Description:

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.

Treatment:

Drug: CMP-CPS-001

Other: Placebo

OTC Heterozygous Participants in Australia

Experimental group

Description:

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.

Treatment:

Drug: CMP-CPS-001

Other: Placebo

OTC Heterozygous Participants in EU

Experimental group

Description:

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.

Treatment:

Drug: CMP-CPS-001

Other: Placebo