PNH
Conditions
Brief summary
This is a single site, single dose escalation study in healthy subject with CAN106. The study is to assess the safety and tolerability of single escalating doses of CAN106; to characterize the PK and PD profile of CAN106; and to evaluate the immunogenicity of CAN106 injection.
Interventions
DRUGCAN106
CAN106 is a selective inhibitor of complement activation, which binds to the complement component C5.
DRUGPlacebo
placebo
Sponsors
CARE Pharma Shanghai Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
21 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
* Subjects must be able to understand and provide informed consent. * Males or females, between 21 and 45 years of age, inclusive; * Body mass index must be within the range of 18.5 to 32.0 kg/m2; * 12-lead electrocardiogram (ECG) within normal limits with no clinically significant abnormalities in the opinion of the Investigator； * Systolic blood pressure ≤140 mmHg and a diastolic blood pressure of ≤ 90 mmHg after 5 minutes with supine rest； * non-pregnancy * meningococcal vaccinations for at least 2 weeks before dosing
Exclusion criteria
* Disease or conditions interfere with participating the trial * Active serious mental illness or psychiatric disorder * clinically relevant abnormal test results in hepatic function * unacceptable CBC lab test * asymptomatic complement deficiency * Any other clinical safety laboratory test * HIV, HBV, HCV positive * Alcohol and drug abuse * etc.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of subjects with dose-limiting toxicity (DLTs) | 6-months after dosing | TEAEs will be categorized as per the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria. a DLT is defined as one subject with a Grade 3 AE or higher, that are assessed as drug-related by the site investigator. |
| Incidence of adverse events (AEs) | 6-months after dosing | An AE is defined as any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. |
| Incidence of severe adverse events (SAEs) | 6-months after dosing | Any untoward medical occurrence that at any dose: * Results in death, * Is life-threatening, * Requires inpatient hospitalization or prolongation of existing hospitalization, * Results in persistent or significant disability/incapacity, or * Is a congenital anomaly/birth defect. (ICH E6 (R2)) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| PK parameters - t1/2 | 6-months after dosing | terminal elimination half-life |
| PD endpoints-free C5 | 6-months after dosing | maximal change from baseline in free C5 concentrations at each of scheduled post baseline assessment time-points (µg/ml); |
| PK parameters - tmax | 6-months after dosing | time to reach maximum of concentration (days) |
| PD endpoints-total C5 | 6-months after dosing | meausure the absolute change from baseline in total C5 concentrations at each of scheduled post baseline assessment time-points (µg/ml); |
| Immunogenicity | 6-months after dosing | Anti-drug Antibody (ADA) titers |
| PD endpoints-CH50 | 6-months after dosing | maximal change from baseline total complement activity (CH50) at each of scheduled post baseline assessment time-points (%); |
| PK parameters-Cmax | 6-months after dosing | peak plasma concentration |
| PK parameters - AUC0-t | 6-months after dosing | Area under the plasma concentration versus time curve to the last visit (AUCt) |
Countries
Singapore
Outcome results
None listed