Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
Primary objective * The tolerability and safety of SAR443809 Secondary * The PK parameters of SAR443809 * The PD activity of SAR443809 * The immunogenicity of SAR443809
Detailed description
Screening: up to 56 days (Day -56 to Day -2). Treatment: 1 day (treatment on Day 1, study observation period from Day -1 to Day 3). Follow-up and end of study: 105 days after IMP administration (follow up visits from Day 5 to Day 78; End of study visit on Day 106). Total study duration for each participant: approximately 23 weeks.
Interventions
DRUGHumanized anti-Factor Bb monoclonal antibody
Pharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration
DRUGPlacebo
Pharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration
Sponsors
Sanofi
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
Having given written informed consent prior to undertaking any study-related procedure
Exclusion criteria
Participants are excluded from the study if any of the following criteria apply: Any participant who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs) | Baseline up to 23 weeks |
| Incidence of potentially Clinical laboratory abnormalities | Baseline up to 23 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to tlast (AUClast) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve extrapolated to infinity (AUC0-∞) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: Terminal half-life associated with the terminal slope (λz) (t1/2z) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: Time corresponding to the last concentration above the limit of quantification (Clast tlast) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: total body clearance of a drug from the plasma calculated by dividing dose by AUC (CL) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for SC administrations: apparent total body clearance of the SC formulation (CL/F) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: Maximum plasma concentration observed (Cmax | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for SC administrations: apparent volume of distribution at steady-state (Vss/F) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for SC administrations: absolute bioavailability (F) | Baseline up to 23 weeks | — |
| Complement alternative pathway activity (Wieslab AP and alternative pathway hemolytic activity [AH50]) | Baseline up to 23 weeks | Ex vivo activity of the alternative pathway of complement in serum using the WIESLAB® Complement System Alternative Pathway kit and a hemolytic assay (AH50) |
| Complement classical pathway activity (Wieslab CP) | Baseline up to 23 weeks | Ex vivo activity of the alternative pathway of complement in serum using the WIESLAB® Complement System Classical Pathway kit |
| Incidence of treatment -emergent Anti-SAR443809 antibodies | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: volume of distribution at steady-state (Vss) | Baseline up to 23 weeks | — |
| PK parameters of SAR443809 for IV and SC administrations: First time to reach Cmax (tmax | Baseline up to 23 weeks | — |
Countries
United States
Outcome results
None listed