Healthy VolunteersPhase 1NCT06247670
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
A Phase 1 study testing CMP-CPS-001 in people with Healthy Volunteers and OTC Deficiency, run by CAMP4 Therapeutics Corporation at 2 sites in 2 countries.
Trial 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 and participants with abnormal heterozygous OTC genotype. Read more about this study
- Who is it for?
- Ages 16 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers, OTC Deficiency
- What is being tested?
- CMP-CPS-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 5 Feb 2024to Dec 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Australia1 site
Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)
Herston, Queensland
About this study
Official title: 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
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.
Read the 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.
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.
- Conditions
- Healthy Volunteers, OTC Deficiency
- Being tested
- CMP-CPS-001, Placebo
- Keywords
- Healthy Volunteers, Antisense Oligonucleotides, Urea Cycle Disorder
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease, Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CMP-CPS-001Drug
CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously.
PlaceboOther
Placebo is 0.9% normal saline solution and will be administered subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose Part | Experimental | Adult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated. Receives: Drug: CMP-CPS-001; Other: Placebo |
| Multiple Ascending Dose Part | Experimental | 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. Receives: Drug: CMP-CPS-001; Other: Placebo |
| OTC Heterozygous Participants in Australia | Experimental | 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. Receives: Drug: CMP-CPS-001; Other: Placebo |
| OTC Heterozygous Participants in EU | Experimental | 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. Receives: Drug: CMP-CPS-001; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001
Measured: Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Secondary outcomes (3)
Plasma PK
Plasma concentration of CMP-CPS-001
Measured: Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Urinary excretion of CMP-CPS-001
Urine concentration of CMP-CPS-001
Measured: 42 days (SAD) or 111 days (MAD, OTC) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
Ureagenesis rate test determination
Measured: Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2024
- Study start5 Feb 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CAMP4 Therapeutics Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gloria Wong, MD, Principal investigator, Q-Pharm Pty LtdMargreet Wagenmakers, Principal investigator, Erasmus Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06247670
- Sponsor's protocol ID
- CPS-101
- Status last verified
- Apr 2026
- First submitted
- 22 Jan 2024
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06247670
- Acronym / sponsor ID
- CPS-101
- Conditions
- Healthy Volunteers, OTC Deficiency
- Keywords
- Healthy Volunteers, Antisense Oligonucleotides, Urea Cycle Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- CAMP4 Therapeutics CorporationIndustry (pharma, biotech, devices)
- Study start
- 5 Feb 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 8 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single Ascending Dose Part | Experimental | Drug: CMP-CPS-001, Other: PlaceboAdult healthy volunteers in 4 cohorts of 12 will receive CMP-CPS-001 or placebo. Four dose levels will be evaluated. |
| Multiple Ascending Dose Part | Experimental | Drug: CMP-CPS-001, Other: PlaceboAdult 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. |
| OTC Heterozygous Participants in Australia | Experimental | Drug: CMP-CPS-001, Other: PlaceboUp 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. |
| OTC Heterozygous Participants in EU | Experimental | Drug: CMP-CPS-001, Other: PlaceboUp 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. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CMP-CPS-001CMP-CPS-001 consists of an antisense oligonucleotide solution that will be administered subcutaneously. | Drug | Multiple Ascending Dose Part, OTC Heterozygous Participants in Australia, OTC Heterozygous Participants in EU, Single Ascending Dose Part |
| PlaceboPlacebo is 0.9% normal saline solution and will be administered subcutaneously. | Other | Multiple Ascending Dose Part, OTC Heterozygous Participants in Australia, OTC Heterozygous Participants in EU, Single Ascending Dose Part |
Primary outcome
Adverse events
Time frame: Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Incidence of adverse events, including dose limiting toxicities, after administration of CMP-CPS-001
3 secondary outcomes
Plasma PK
Time frame: Pre-dose (Day 1) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Urinary excretion of CMP-CPS-001
Time frame: 42 days (SAD) or 111 days (MAD, OTC) after dosing
Pharmacodynamic effect of CMP-CPS-001 on ureagenesis
Time frame: Run-in (Day -7) until 42 days (SAD) or 112 days (MAD, OTC) after dosing
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gloria Wong, MD principal investigator
Q-Pharm Pty Ltd
Margreet Wagenmakers principal investigator
Erasmus Medical Center
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CMP-CPS-001 in people with Healthy Volunteers and OTC Deficiency, run by CAMP4 Therapeutics Corporation at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: Screening until 42 days (SAD) or 112 days (MAD, OTC) after dosing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026.