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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:
Disease Driven by Complement Activation 0 0
Condition category
Condition code

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

Experimental: Part A [Single ascending dose (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 [Multiple ascending dose (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 to 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
Number of participants with treatment emergent adverse events (TEAEs), adverse events of special interests (AESIs), and serious adverse events (SAEs)
Timepoint [1] 0 0
Part A (SAD): Up to 105 days; Part B (MAD): Up to 118 days
Primary outcome [2] 0 0
Percentages of participants with TEAEs, AESIs, and SAEs
Timepoint [2] 0 0
Part A (SAD): Up to 105 days; Part B (MAD): Up to 118 days
Primary outcome [3] 0 0
The Number of Clinically Significant Changes from Baseline in Clinical Laboratory Tests Reported as AE
Timepoint [3] 0 0
Baseline and up to 69 days
Primary outcome [4] 0 0
Number of participants with vital signs out of normal range
Timepoint [4] 0 0
Baseline and up to 69 days
Primary outcome [5] 0 0
Change from Baseline in corrected QT interval using Fridericia's formula (QTcF) values of triplicate electrocardiograms
Timepoint [5] 0 0
Baseline and up to 69 days
Primary outcome [6] 0 0
Absolute values of QTcF on electrocardiograms
Timepoint [6] 0 0
Baseline and up to 69 days
Primary outcome [7] 0 0
Number of participants with abnormal electrocardiogram findings
Timepoint [7] 0 0
Baseline and up to 69 days
Secondary outcome [1] 0 0
Part A (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 (MAD): Maximum concentration (Cmax)
Timepoint [8] 0 0
Up to 69 days
Secondary outcome [9] 0 0
Part B (MAD): Time to reach maximum concentration (Tmax)
Timepoint [9] 0 0
Up to 69 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 69 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 69 days
Secondary outcome [12] 0 0
Part B (MAD): Lowest concentration prior to dosing (Ctrough)
Timepoint [12] 0 0
Up to 69 days
Secondary outcome [13] 0 0
Part B (MAD): Total systemic clearance (CL)
Timepoint [13] 0 0
Up to 69 days
Secondary outcome [14] 0 0
Part B (MAD): Volume of distribution at steady state (Vss)
Timepoint [14] 0 0
Up to 69 days
Secondary outcome [15] 0 0
Part A (SAD) and Part B (MAD): Percent change from Baseline in pharmacodynamic (PD) parameters
Timepoint [15] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [16] 0 0
Part A (SAD) and Part B (MAD): Maximum percent change from Baseline (Emax) in PD parameters
Timepoint [16] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [17] 0 0
Part A (SAD) and Part B (MAD): Time to maximum percent change from Baseline (TEmax) in PD parameters
Timepoint [17] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [18] 0 0
Part A (SAD) and Part B (MAD): Time below Baseline in PD parameters
Timepoint [18] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [19] 0 0
Part A (SAD) and Part B (MAD): AUC below Baseline in PD parameters
Timepoint [19] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [20] 0 0
Part A (SAD) and Part B (MAD): Number of serum samples with Positive antidrug antibodies (ADAs) binding to CSL040
Timepoint [20] 0 0
Up to 56 days (Part A) and up to 69 days (Part B)
Secondary outcome [21] 0 0
Part A (SAD) and Part B (MAD): Number of participants with presence of treatment-emergent ADAs
Timepoint [21] 0 0
Up to 56 days (Part A) and up to 69 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 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
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. 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

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
30/04/2025
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
+1 610-878-4697
Fax 0 0
Email 0 0
clinicaltrials@cslbehring.com
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 requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

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.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
Available to whom?
Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.

An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.

The requesting party must execute an appropriate data sharing agreement before IPD will be made 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