Butyrylcholinesterase Deficiency
Conditions
Keywords
Butyrylcholinesterase deficiency, tolerability, safety, AZD8848, pharmacokinetics, pharmacodynamics, BChE deficient subjects and matched control subject
Brief summary
The purpose of this study is to investigate the safety and tolerability of AZD8848 in Butyrylcholinesterase deficient subjects in comparison with sex and age matched control subjects.
Detailed description
An Open, Non-controlled, Non-randomised, Parallel-group Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of AZD 8848 Administered Intranasally to Male and Female Butyrylcholinesterase deficient Subjects and to Sex and Age matched Controls
Interventions
DRUGAZD8848
Nasal spray solution, intranasal, single ascending doses, 1.4 - 60 μg
Sponsors
AstraZeneca
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
Yes
Inclusion criteria
* BChE deficient subjects: The half-live of AZD8848 in plasma should be more than 20 minutes in an in vitro screening test * Matched control subject: The half-live of AZD8848 in plasma should be less than 2 minutes in an in vitro screening test
Exclusion criteria
* Any clinically relevant abnormal findings in physical examination, laboratory assessments, vital signs or ECG * Present or medical history of cardiovascular disease which, in the opinion of the investigator, may either put subject at risk because of participation in the study or influence the result of the study or the subject's ability to participate in the study * Family history of autoimmune disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability will be assessed by a panel of measurements including adverse events (AEs), vital signs, ECG, laboratory variables, physical examination, and clinical inspection of the nose | Immediately prior to administration of the IP (Day 0) |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetics (concentration of AZD8848 and metabolite in plasma and urine) | Prior to administration of the IP (Day 0) and repeated assessments during the first 48h. |
| Pharmacodynamics (IL-1Ra in plasma) | Prior to administration of the IP (Day 0) and repeated assessments during the first 48h. |
Countries
Denmark
Outcome results
None listed