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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05937581




Registration number
NCT05937581
Ethics application status
Date submitted
18/05/2023
Date registered
10/07/2023

Titles & IDs
Public title
First-In-Human Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Escalating Single And Multiple Doses Of CSL040 In Healthy Subjects
Scientific title
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
Secondary ID [1] 0 0
CSL040_1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Complement Deficiencies 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - CSL040
Treatment: Drugs - Placebo

Experimental: Part A (SAD): CSL040 (minimum dose) - Single Intravenous (IV) administration

Experimental: Part A (SAD): CSL040 (lower dose) - Single IV Administration

Experimental: Part A (SAD): CSL040 (low dose) - Single IV Administration

Experimental: Part A (SAD): CSL040 (medium dose) - Single IV Administration

Experimental: Part A (SAD): CSL040 (medium-high dose) - Single IV Administration

Experimental: Part A (SAD): CSL040 (maximum dose) - Single IV Administration

Placebo comparator: Part A (SAD): Placebo - Single IV Administration

Experimental: Part B (MAD): CSL040 (minimum dose) - IV Administration not to exceed 5 doses over 14 days)

Experimental: Part B (MAD): CSL040 (medium dose) - IV Administration not to exceed 5 doses over 14 days

Experimental: Part B (MAD): CSL040 (high dose) - IV Administration not to exceed 5 doses over 14 days

Placebo comparator: Part B (MAD): Placebo - IV Administration not go exceed 5 doses over 14 days


Treatment: Drugs: CSL040
IV Administration

Treatment: Drugs: Placebo
0.9% w/v NaCI, IV Administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency counts for subjects with treatment emergent adverse events (TEAEs) leading to withdrawal or discontinuation of investigational product
Timepoint [1] 0 0
Up to 10 weeks
Primary outcome [2] 0 0
Percentages for subjects with treatment emergent TEAEs leading to withdrawal or discontinuation of investigational product
Timepoint [2] 0 0
Up to 10 weeks
Primary outcome [3] 0 0
Change from Baseline in tympanic temperature
Timepoint [3] 0 0
Up to 10 weeks
Primary outcome [4] 0 0
Change from Baseline in pulse rate
Timepoint [4] 0 0
Up to 10 weeks
Primary outcome [5] 0 0
Change from Baseline in blood pressure
Timepoint [5] 0 0
Up to 10 weeks
Primary outcome [6] 0 0
Change from Baseline in leukocytes (white blood cell count)
Timepoint [6] 0 0
Up to 10 weeks
Primary outcome [7] 0 0
Change from Baseline of hemoglobin values
Timepoint [7] 0 0
Up to 10 weeks
Primary outcome [8] 0 0
Change from Baseline of C-reactive protein (CRP)
Timepoint [8] 0 0
Up to 10 weeks
Primary outcome [9] 0 0
Change from Baseline in Creatinine levels
Timepoint [9] 0 0
Up to 10 weeks
Primary outcome [10] 0 0
Change from Baseline above the upper limit of normal (ULN) for alanine aminotransaminase (ALT)
Timepoint [10] 0 0
Up to 10 weeks
Primary outcome [11] 0 0
Change from Baseline above the upper limit of normal (ULN) for aspartate aminotransaminase (AST)
Timepoint [11] 0 0
Up to 10 weeks
Primary outcome [12] 0 0
Change from Baseline in corrected QT interval using Fridericia's formula (QTcF) values of triplicate electrocardiograms
Timepoint [12] 0 0
Up to 10 weeks
Secondary outcome [1] 0 0
Part A (Single Ascending Dose [SAD]): Maximum concentration (Cmax)
Timepoint [1] 0 0
Up to 56 Days
Secondary outcome [2] 0 0
Part A (SAD): Time to reach maximum concentration (Tmax)
Timepoint [2] 0 0
Up to 56 Days
Secondary outcome [3] 0 0
Part A (SAD): Time to Area under the concentration-time curve from time 0 to the last measurable concentration (AUC0-last)
Timepoint [3] 0 0
Up to 56 Days
Secondary outcome [4] 0 0
Part A (SAD): Area under the concentration-time curve from time 0 to infinity (AUC0-infinity)
Timepoint [4] 0 0
Up to 56 Days
Secondary outcome [5] 0 0
Part A (SAD): Total systemic clearance (CL)
Timepoint [5] 0 0
Up to 56 Days
Secondary outcome [6] 0 0
Part A (SAD): Volume of distribution (V)
Timepoint [6] 0 0
Up to 56 Days
Secondary outcome [7] 0 0
Part A (SAD): Terminal elimination half-life (T1/2)
Timepoint [7] 0 0
Up to 56 Days
Secondary outcome [8] 0 0
Part B (Multiple Ascending Dose [MAD]): Maximum concentration (Cmax)
Timepoint [8] 0 0
Up to 70 Days
Secondary outcome [9] 0 0
Part B (MAD): Time to reach maximum concentration (Tmax)
Timepoint [9] 0 0
Up to 70 Days
Secondary outcome [10] 0 0
Part B (MAD): Time to Area under the concentration-time curve in 1 dosing interval (AUCtau)
Timepoint [10] 0 0
Up to 70 Days
Secondary outcome [11] 0 0
Part B (MAD): Accumulation index (accumulation ratio determined by the ratio of steady state AUCtau to single dose AUCtau
Timepoint [11] 0 0
Up to 70 Days
Secondary outcome [12] 0 0
Part B (MAD): Lowest concentration prior to dosing (Ctrough)
Timepoint [12] 0 0
Up to 70 Days
Secondary outcome [13] 0 0
Part B (MAD): Total systemic clearance (CL)
Timepoint [13] 0 0
Up to 70 Days
Secondary outcome [14] 0 0
Part B (MAD): Volume of distribution at steady state (Vss)
Timepoint [14] 0 0
Up to 70 Days
Secondary outcome [15] 0 0
Part A (SAD) and Part B (MAD): Maximum percent change from Baseline (Emax)
Timepoint [15] 0 0
Up to 56 Days (Part A) and up to 70 days (Part B)
Secondary outcome [16] 0 0
Part A (SAD) and Part B (MAD): Time to maximum percent change from Baseline (TEmax)
Timepoint [16] 0 0
Up to 56 Days (Part A) and up to 70 days (Part B)
Secondary outcome [17] 0 0
Part A (SAD) and Part B (MAD): Time to return to baseline
Timepoint [17] 0 0
Up to 56 Days (Part A) and up to 70 days (Part B)
Secondary outcome [18] 0 0
Part A (SAD) and Part B (MAD): Presence of treatment-emergent antidrug antibodies after administration of CSL040
Timepoint [18] 0 0
Up to 56 Days (Part A) and up to 70 days (Part B)

Eligibility
Key inclusion criteria
1. Male or female 18 to 64 years of age, inclusive, at Screening
2. Body weight in the range of = 50 kg and = 100 kg, with a body mass index of = 18 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 vaccination against meningococcal disease according to local requirements or willing to receive meningococcal vaccination aligned with local guidelines (vaccination with recombinant meningococcal B vaccine against B serogroup [Men B] and quadrivalent meningococcal conjugate vaccine against A, C, W, and Y serogroups [Men ACWY]), 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
Minimum age
18 Years
Maximum age
64 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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. Other than the required meningococcal vaccines, any live vaccinations within the last 90 days before and during the study and / or up to 90 days after the last administration of the investigational product
3. A positive test result for hepatitis B surface antigen (HBsAg), hepatitis B core antigen, hepatitis B surface antibody, 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
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 infections, or any infection which makes the participation in this study of healthy subjects 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 the CSL040 or any of the excipients or other monoclonal antibodies
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) 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 the vaccination as required per protocol) within the 21 days before 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 mL of blood within 3 months, or donated plasma within 7 days, before admission to the unit and for 5 half-lives 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2025
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Nucleus Network Pty Ltd - Herston
Recruitment postcode(s) [1] 0 0
4006 - Herston

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
CSLBehring LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Registration Coordinator
Address 0 0
Country 0 0
Phone 0 0
1-610-878-4000
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at [email protected].
When will data be available (start and end dates)?
Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and/or the primary publication is available.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05937581