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A Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Phase 1 Study of CMP-CPS-001 in Healthy Volunteers and Participants with Abnormal Heterozygous Ornithine Transcarbamylase (OTC) Genotype
EU CTIS ID: 2025-520882-37-00
What this study is testing
1. To evaluate the safety and tolerability of CMP-CPS-001 administered SC to study participants
- Human Pharmacology (Phase I)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Written consent obtained from the study participant and documented.
- 2. BMI ≥18.0 and ≤32 kg/m2 at screening, and ≤110 kg.
- 3. Module 1 Part C and Module 2: Participants must be female and must have confirmed heterozygous OTC genotype.
- 4. Module 1: Participants 18 to <65 years of age inclusive at time of informed consent.
- 5. Module 2: Participants 16 to <65 years of age inclusive at time of informed consent.
You likely can't join if
- 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
- 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.
- 3. History of intolerance to SC injection or relevant abdominal scarring.
- 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.
- 5. Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus (HCV) antibody.
- 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.
See the full eligibility criteria
- 1. Written consent obtained from the study participant and documented.
- 2. BMI ≥18.0 and ≤32 kg/m2 at screening, and ≤110 kg.
- 3. Module 1 Part C and Module 2: Participants must be female and must have confirmed heterozygous OTC genotype.
- 4. Module 1: Participants 18 to <65 years of age inclusive at time of informed consent.
- 5. Module 2: Participants 16 to <65 years of age inclusive at time of informed consent.
- 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
- 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.
- 3. History of intolerance to SC injection or relevant abdominal scarring.
- 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.
- 5. Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus (HCV) antibody.
- 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.
- 7. Any other safety laboratory result considered clinically significant and unacceptable by the Investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.