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

2025-520882-37-00 Protocol CPS-101 Human pharmacology (Phase I) - First administration to humans Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites · Protocol CPS-101

Overview

Sponsor-declared trial summary

Phase Human pharmacology (Phase I) - First administration to humans
Status Authorised, recruitment pending
Participants planned 144
Countries 1
Sites 1

Urea cycle disorders (UCD)

1. To evaluate the safety and tolerability of CMP-CPS-001 administered SC to study participants

Key facts

Sponsor
Camp4 Therapeutics Corp.
Participant type
Pediatric, Healthy volunteers, Patients
Age range
0-17 years, 18-64 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Nutritional and Metabolic Diseases [C18]
Decision date (initial)
2025-08-09
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
CAMP4 Therapeutics Corporation

External identifiers

EU CT number
2025-520882-37-00
ClinicalTrials.gov
NCT06247670

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Others, Safety

1. To evaluate the safety and tolerability of CMP-CPS-001 administered SC to study participants

Secondary objectives 3

  1. 1. To characterize the plasma PK of CMP-CPS-001
  2. 2. To determine the urinary excretion of CMP-CPS-001
  3. 3. To evaluate the effect of CMP-CPS-001 on ureagenesis

Conditions and MedDRA coding

Urea cycle disorders (UCD)

VersionLevelCodeTermSystem organ class
20.1 PT 10080020 Urea cycle disorder 100000004850

Study design 4 periods

#TitleAllocationBlindingRoles blindedArms
1 Module 1: Part A (SAD) in adult healthy participants
A single dose of CMP-CPS-001 or placebo in up to 72 healthy participants (4 cohorts of 12 and 2 optional cohorts of 12).
 Randomised Controlled Double [{"id":134672,"code":2,"name":"Investigator"},{"id":134673,"code":1,"name":"Subject"}] CMP-CPS-001: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
Placebo: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
2 Module 1: Part B (MAD) in adult healthy participants
3 doses of either CMP-CPS-001 or placebo once every 4 weeks (q4w) in up to 48 healthy participants (4 cohorts of 12 each).
 Randomised Controlled Double [{"id":134676,"code":2,"name":"Investigator"},{"id":134675,"code":1,"name":"Subject"}] CMP-CPS-001: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
Placebo: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
3 Module 1: Part C (MAD) in female participants with an abnormal heterozygous OTC genotype
3 doses of either CMP-CPS-001 or placebo once every 4 weeks (q4w) in up to 12 clinically healthy participants.
 Randomised Controlled Double [{"id":134679,"code":1,"name":"Subject"},{"id":134678,"code":2,"name":"Investigator"}] CMP-CPS-001: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
Placebo: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
4 Module 2 in female participants with an abnormal heterozygous OTC genotype
3 doses of either CMP-CPS-001 or placebo once every 4 weeks (q4w) in up to 12 clinically healthy participants.
 Randomised Controlled Double [{"id":134681,"code":2,"name":"Investigator"},{"id":134682,"code":1,"name":"Subject"}] CMP-CPS-001: Randomization ratio is 3:1 (CMP-CPS-001:placebo)
Placebo: Randomization ratio is 3:1 (CMP-CPS-001:placebo)

Regulatory references

Scientific advice from competent authorities
Food And Drug Administration
Plan to share IPD
No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

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

Exclusion criteria 7

  1. 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. 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. 3. History of intolerance to SC injection or relevant abdominal scarring.
  4. 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. 5. Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus (HCV) antibody.
  6. 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. 7. Any other safety laboratory result considered clinically significant and unacceptable by the Investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

  1. 1. Incidence of AEs
  2. 2. Vital signs
  3. 3. ECG
  4. 4. Clinical laboratory assessment
  5. 5. Physical examination

Secondary endpoints 4

  1. 1. Plasma concentration of CO-5122, the active ingredient of CMP-CPS-001
  2. 2. Calculated PK parameters
  3. 3. Urine concentration of CO-5122, the active ingredient of CMP-CPS-001
  4. 4. Ureagenesis rate test determinations

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

CMP-CPS-001

PRD12302941 · Product

Active substance
CO-5122
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS
Authorisation status
Not Authorised
MA holder
CAMP4 THERAPEUTICS CORP.
Paediatric formulation
No
Orphan designation
No

Placebo 1

Sodium Chloride for injection

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Auxiliary 2

[13C]Sodium Acetate

PRD12303080 · Product

Active substance
Sodium Acetate (1-13C)
Substance synonyms
[1-13C]sodium acetate
Pharmaceutical form
POWDER FOR ORAL SOLUTION
Route of administration
ORAL
Authorisation status
Not Authorised
MA holder
CAMP4 THERAPEUTICS CORP.
Paediatric formulation
No
Orphan designation
No

15NH4Cl

PRD12303081 · Product

Active substance
Ammonium (15N) Chloride
Substance synonyms
15N Ammonium chloride
Pharmaceutical form
POWDER FOR ORAL SOLUTION
Route of administration
ORAL
Authorisation status
Not Authorised
MA holder
CAMP4 THERAPEUTICS CORP.
Paediatric formulation
No
Orphan designation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Camp4 Therapeutics Corp.

Sponsor organisation
Camp4 Therapeutics Corp.
Address
1 Kendall Square
City
Cambridge
Postcode
02139-1562
Country
United States

Scientific contact point

Organisation
Camp4 Therapeutics Corp.
Contact name
Yuri Maricich

Public contact point

Organisation
Camp4 Therapeutics Corp.
Contact name
Yuri Maricich

Third parties 8

OrganisationCity, countryDuties
Biobridges LLC
ORG-100054014
 Morrisville, United States Code 11
Altasciences Compagnie Inc.
ORG-100037610
 Laval, Canada Laboratory analysis
Metabolic Solutions LLC
ORG-100054033
 Nashua, United States Laboratory analysis
Amsterdam UMC Research B.V.
ORG-100053868
 Amsterdam, Netherlands Laboratory analysis
Greenphire LLC
ORG-100041621
 King Of Prussia, United States Other
Eresearchtechnology Inc.
ORG-100013039
 Philadelphia, United States Other
Rittia Medical Writing & Consulting LLC
ORG-100054045
 Acton, United States Code 11
Icon Clinical Research Limited
ORG-100008322
 Dublin 18, Ireland On site monitoring, Code 10, Code 11, Code 12, Code 5, Data management, Code 8

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Netherlands Authorised, recruitment pending 12 1
Rest of world
Australia
 — 132 —

Investigational sites

Netherlands

1 site · Authorised, recruitment pending
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Internal Medicine, Dr. Molewaterplein 40, 3015 GD, Rotterdam

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-04-30 Netherlands Acceptable
2025-08-09
 2025-08-09

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