First-In-Human Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Escalating Single And Multiple Doses Of CSL040 In Healthy Subjects

NCT05937581 | Completed Phase 1

• 1 other identifier: CSL040_1001
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 1

Brief Summary

First-In-Human Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Escalating Single And Multiple Doses Of CSL040 In Healthy Subjects

Conditions

Complement-mediated Disorders

Keywords

Complement

Outcome Measures

Primary Outcomes (7)

• Number of participants with treatment emergent adverse events (TEAEs), adverse events of special interests (AESIs), and serious adverse events (SAEs)

  Part A (SAD): Up to 105 days; Part B (MAD): Up to 174 days

• Percentages of participants with TEAEs, AESIs, and SAEs

  Part A (SAD): Up to 105 days; Part B (MAD): Up to 174 days

• The Number of Clinically Significant Changes from Baseline in Clinical Laboratory Tests Reported as AE

  Clinical laboratory tests include hematology, biochemistry, coagulation, and urinalysis collected during the study. The investigator determines if the changes in laboratory test results are clinically significant.

  Baseline and up to 69 days

• Number of participants with vital signs out of normal range

  Blood pressure (systolic and diastolic), pulse rate, respiratory rate and tympanic temperature will be assessed.

  Baseline and up to 69 days

• Change from Baseline in corrected QT interval using Fridericia's formula (QTcF) values of triplicate electrocardiograms

  Baseline and up to 69 days

• Absolute values of QTcF on electrocardiograms

  Baseline and up to 69 days

• Number of participants with abnormal electrocardiogram findings

  Baseline and up to 69 days

Secondary Outcomes (21)

• Part A (SAD): Maximum concentration (Cmax)

  Up to 56 days

• Part A (SAD): Time to reach maximum concentration (Tmax)

  Up to 56 days

• Part A (SAD): Time to Area under the concentration-time curve from time 0 to the last measurable concentration (AUC0-last)

  Up to 56 days

• Part A (SAD): Area under the concentration-time curve from time 0 to infinity (AUC0-infinity)

  Up to 56 days

• Part A (SAD): Total systemic clearance (CL)

  Up to 56 days

Study Arms (11)

Part A [Single ascending dose (SAD)]: CSL040 (minimum dose)

EXPERIMENTAL

Single Intravenous (IV) Administration

Drug: CSL040

Part A (SAD): CSL040 (lower dose)

EXPERIMENTAL

Single IV Administration

Drug: CSL040

Part A (SAD): CSL040 (low dose)

EXPERIMENTAL

Single IV Administration

Drug: CSL040

Part A (SAD): CSL040 (medium dose)

EXPERIMENTAL

Single IV Administration

Drug: CSL040

Part A (SAD): CSL040 (medium-high dose)

EXPERIMENTAL

Single IV Administration

Drug: CSL040

Part A (SAD): CSL040 (maximum dose)

EXPERIMENTAL

Single IV Administration

Drug: CSL040

Part A (SAD): Placebo

PLACEBO COMPARATOR

Single IV Administration

Drug: Placebo

Part B [Multiple ascending dose (MAD)]: CSL040 (minimum dose)

EXPERIMENTAL

IV Administration not to exceed 5 doses over 14 days

Drug: CSL040

Part B (MAD): CSL040 (medium dose)

EXPERIMENTAL

IV Administration not to exceed 5 doses over 14 days

Drug: CSL040

Part B (MAD): CSL040 (high dose)

EXPERIMENTAL

IV Administration not to exceed 5 doses over 14 days

Drug: CSL040

Part B (MAD): Placebo

PLACEBO COMPARATOR

IV Administration not to exceed 5 doses over 14 days

Drug: Placebo

Interventions

CSL040 DRUG

IV Administration

Part A (SAD): CSL040 (low dose); Part A (SAD): CSL040 (lower dose); Part A (SAD): CSL040 (maximum dose); Part A (SAD): CSL040 (medium dose); Part A (SAD): CSL040 (medium-high dose); Part A [Single ascending dose (SAD)]: CSL040 (minimum dose); Part B (MAD): CSL040 (high dose); Part B (MAD): CSL040 (medium dose); Part B [Multiple ascending dose (MAD)]: CSL040 (minimum dose)

Placebo DRUG

0.9% w/v NaCI, IV Administration

Part A (SAD): Placebo; Part B (MAD): Placebo

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female 18 to 64 years of age, inclusive, at Screening
• 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
• Judged as healthy by an Investigator after completion of a comprehensive clinical assessment
• Capable of providing written informed consent and willing and able to adhere to all protocol requirements
• Can understand the nature, scope, and possible consequences of the study and able to comply with study procedures, restrictions, and requirements
• \. 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 per the Australian Immunisation Handbook
• 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
• 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
• 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

You may not qualify if:

• 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
• \. 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
• A positive test result for any of the following: hepatitis B screening, hepatitis C virus antibody, or human immunodeficiency virus-1/2 antibody
• History concerning for a N. meningitidis infection
• History of allergy or intolerance to Penicillin V, as well as to potential backup medications including azithromycin, ciprofloxacin, and ceftriaxone
• History of unexplained, recurrent infection, life-threatening infection, or history that suggests any immunodeficiency (functional immunodeficiency), including asplenia / functional asplenia
• Infection requiring treatment with systemic antibiotics (IV and / or oral administration for more than 3 days) within the last 90 days prior to dosing
• Clinical evidence of current active serious infection, including any localized infection, or any infection which makes participation in this study unacceptably high risk
• Blood pressure or pulse rate measurements outside the normal range for the subject's age and assessed as clinically significant
• 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.
• 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
• A positive test result for drugs of abuse (including alcohol) and cotinine at Screening and / or Day -1.
• Weekly alcohol intake of \> 10 units for females and \> 14 units for males during the 3 months before Day -1.
• Any values above the upper limit of normal (ULN) for alanine aminotransaminase (ALT) or aspartate aminotransaminase (AST), or bilirubin test result
• 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

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (1)

Nucleus Network Pty Ltd

Herston, Queensland, 4006, Australia

Location

Study Officials

• Study Director

  CSLBehring LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2023
• First Posted: July 10, 2023
• Study Start: September 28, 2023
• Primary Completion: December 4, 2025
• Study Completion: December 4, 2025
• Last Updated: February 3, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Locations

AU (1)