CO-5122 for Urea cycle disorder|Ornithine transcarbamylase deficiency

Inclusion criteria

• {"criterion_text":"- 1. Written consent obtained from the study participant and documented.\n- 2. BMI ≥18.0 and ≤32 kg/m2 at screening, and ≤110 kg.\n- 3. Module 1 Part C and Module 2: Participants must be female and must have confirmed heterozygous OTC genotype.\n- 4. Module 1: Participants 18 to <65 years of age inclusive at time of informed consent.\n- 5. Module 2: Participants 16 to <65 years of age inclusive at time of informed consent."}

Exclusion criteria

• {"criterion_text":"- 1. 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\n- 2. Clinically relevant illness or infection within 7 days before the first dose of study drug that would risk integrity of the study, its data and/or participation, in the opinion of the Investigator.\n- 3. History of intolerance to SC injection or relevant abdominal scarring.\n- 4. Laboratory results outside of the normal ranges at screening and judged as clinically relevant by the Investigator for liver function, kidney function, and platelets.\n- 5. Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus (HCV) antibody.\n- 6. History of multiple drug allergies, history of serious anaphylaxis, or history of allergic reaction to oligonucleotides, GalNAc, polyethylene glycol, compound used for ureagenesis rate test, or other excipients.\n- 7. Any other safety laboratory result considered clinically significant and unacceptable by the Investigator."}

Primary endpoints

• {"endpoint_text":"- 1. Incidence of AEs","definition_or_measurement_approach":""}
• {"endpoint_text":"- 2. Vital signs","definition_or_measurement_approach":""}
• {"endpoint_text":"- 3. ECG","definition_or_measurement_approach":""}
• {"endpoint_text":"- 4. Clinical laboratory assessment","definition_or_measurement_approach":""}
• {"endpoint_text":"- 5. Physical examination","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- 1. Plasma concentration of CO-5122, the active ingredient of CMP-CPS-001","definition_or_measurement_approach":""}
• {"endpoint_text":"- 2. Calculated PK parameters","definition_or_measurement_approach":""}
• {"endpoint_text":"- 3. Urine concentration of CO-5122, the active ingredient of CMP-CPS-001","definition_or_measurement_approach":""}
• {"endpoint_text":"- 4. Ureagenesis rate test determinations","definition_or_measurement_approach":""}

Netherlands

Earliest CTIS Part Ii Submission Date

07-07-2025

Latest Decision Or Authorization Date

09-08-2025

Processing Time Days

33

Number Of Sites

1

Number Of Participants

12

Primary sponsor

Full Name

Camp4 Therapeutics Corp.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Codes: 1,10,11,12,5,6,8

Third parties

• {"country":"United States","full_name":"Biobridges LLC","duties_or_roles":"11","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Altasciences Compagnie Inc.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Metabolic Solutions LLC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Amsterdam UMC Research B.V.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient reimbursements and Travel","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG readings","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Rittia Medical Writing & Consulting LLC","duties_or_roles":"11","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"1,10,11,12,5,6,8","organisation_type":"Pharmaceutical company"}