Evaluating the safety and effects of CSL040 in healthy individuals

A Phase 1, Double-Blind (Sponsor-Unblinded), Placebo-Controlled, Dose Escalation Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Single And Multiple Doses Of CSL040 In Healthy Adult Subjects

This study is testing a new drug called CSL040 in healthy people to see how safe it is and how it works in the body.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 18 Years to 64 Years
• Sex: All
• Sponsor: CSL Behring (industry)
• Locations: 1 site (Herston, Queensland)
• Trial ID: NCT05937581 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of escalating doses of CSL040 in healthy subjects. Participants will receive either CSL040 or a placebo to determine how the drug behaves in the body and its potential effects. The study will involve both single and multiple dosing regimens to gather comprehensive data on the drug's profile. The findings will help inform future studies and potential therapeutic applications for conditions driven by complement activation.

Who should consider this trial

Good fit: Ideal candidates are healthy males and females aged 18 to 64 who meet specific health criteria.

Not a fit: Patients with pre-existing health conditions or those outside the specified age and weight range may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to new treatment options for diseases related to complement activation.

How similar studies have performed: While this is a first-in-human study, similar approaches in evaluating new therapies targeting complement activation have shown promise in other contexts.

Eligibility criteria

Inclusion Criteria:

1. Male or female 18 to 64 years of age, inclusive, at Screening
2. Body weight in the range of greater than or equal to (≥) 50 kg and less than or equal to (≤) 100 kilogram (kg) , with a body mass index of ≥ 18 kilogram per meter square (kg/m2) and ≤ 30 kg/m2, at Screening
3. Judged as healthy by an Investigator after completion of a comprehensive clinical assessment
4. Capable of providing written informed consent and willing and able to adhere to all protocol requirements
5. Can understand the nature, scope, and possible consequences of the study and able to comply with study procedures, restrictions, and requirements
6. Able to provide proof of adequate vaccination (as determined by the Investigator) against meningococcal disease, including vaccination against meningococcal serogroup B and meningococcal serogroups A, C, W, and Y OR be willing to receive additional vaccinations against these serogroups with the first dose at least 21 days before the first dose of CSL040
7. Continuous nonsmoker who has not used nicotine- and tobacco-containing products for at least 30 days prior to the first dosing based on urine cotinine testing at Screening and Day-1
8. Able to provide proof of adequate vaccination (as determined by the Investigator) against Haemophilus influenzae type b, Pneumococcus OR be willing to receive additional vaccinations against these pathogens with the first dose at least 21 days before the first dose of CSL040
9. Able to provide proof of adequate vaccination (as determined by the Investigator) against Severe Acute Respiratory Syndrome Coronavirus 2 (SARS_CoV-2) OR be willing to receive additional vaccination(s) to achieve adequate vaccination status at least 14 days before the first dose of CSL040. If there is proof of a recent SARS-CoV-2 infection (as determined by the Investigator) within 90 days of the first dose of CSL040, the requirement for a vaccination will follow the current local clinical practice

Exclusion Criteria:

1. Any individual at high risk of exposure to Neisseria meningitidis, including, but not limited to, health care workers, doctors, nurses, students working in a clinical setting, laboratory workers with exposure to N. meningitidis, individuals residing in a dormitory setting (eg, military workers), and childcare workers
2. Vaccination with any live replication-competent vaccine 90 days before Day 1 or planned vaccination with the same within 90 days after the last administration of CSL040
3. A positive test result for any of the following: hepatitis B screening, hepatitis C virus antibody, or human immunodeficiency virus-1/2 antibody
4. History concerning for a N. meningitidis infection
5. History of allergy or intolerance to Penicillin V, as well as to potential backup medications including azithromycin, ciprofloxacin, and ceftriaxone
6. History of unexplained, recurrent infection, life-threatening infection, or history that suggests any immunodeficiency (functional immunodeficiency), including asplenia / functional asplenia
7. Infection requiring treatment with systemic antibiotics (IV and / or oral administration for more than 3 days) within the last 90 days prior to dosing
8. Clinical evidence of current active serious infection, including any localized infection, or any infection which makes participation in this study unacceptably high risk
9. Blood pressure or pulse rate measurements outside the normal range for the subject's age and assessed as clinically significant
10. Known history of severe hypersensitivity reactions or suspected hypersensitivity to CSL040 or any excipients including polysorbate 80, monoclonal antibodies, or any documented history of a severe allergic reaction (in the opinion of the Investigator), angioedema, or anaphylaxis to food or any other drugs.
11. Subject has any condition that may compromise their safety or compliance, impede successful conduct of the study, interfere with interpretation of the results or would otherwise render the subject unsuitable for participation in the study
12. A positive test result for drugs of abuse (including alcohol) and cotinine at Screening and / or Day -1.
13. Weekly alcohol intake of \> 10 units for females and \> 14 units for males during the 3 months before Day -1.
14. Any values above the upper limit of normal (ULN) for alanine aminotransaminase (ALT) or aspartate aminotransaminase (AST), or bilirubin test result
15. Use of prescription or over-the-counter medication, herbal and dietary supplements, and vitamins and minerals (except any vaccinations or other medications required/permitted as per protocol) within the 21 days before first administration of investigational product
16. Female subject of childbearing potential or fertile male subject who are neither using nor willing to use a highly effective method of contraception
17. Pregnant, lactating, or breastfeeding
18. Donation or loss of more than 500 milliLiter (mL) of blood within 3 months, or donation of plasma within 7 days, before admission to the unit or plans to donate blood or plasma and for 5 half-lives of the last dose of CSL040 or until the end of the study, whichever is longer
19. Any planned surgical procedures during the study period
20. Participation in any other investigational product study in which receipt of an investigational product occurred within 5 half-lives or 28 days (whichever is longer) before dosing of investigational product, or participation in more than 4 clinical studies involving administration of an investigational product within the last 12 months before Screening
21. Subject who met all eligibility criteria but was not needed (ie, alternate subjects). Alternate subjects are eligible to participate in subsequent cohorts
22. Prior dosing with CSL040

Where this trial is running

Herston, Queensland

• Nucleus Network Pty Ltd — Herston, Queensland, Australia (RECRUITING)

Study contacts

• Study coordinator: Trial Registration Coordinator

Conditions: Disease Driven by Complement Activation, Complement