Clinical trial NCT01205867: Study to Investigate the Safety and Tolerability of AZD8848 in Butyrylcholinesterase Deficient Subjects
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Butyrylcholinesterase Deficiency
Keywords: Butyrylcholinesterase deficiency
tolerability
safety
AZD8848
pharmacokinetics
pharmacodynamics
BChE deficient subjects and matched control subject
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2010-09-21
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: 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
timeFrame: Immediately prior to administration of the IP (Day 0)
measure: 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
timeFrame: Repeated assessments during Day 1.
measure: 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
timeFrame: Repeated assessments during Day 2.
Secondary outcomes:
measure: Pharmacokinetics (concentration of AZD8848 and metabolite in plasma and urine)
timeFrame: Prior to administration of the IP (Day 0) and repeated assessments during the first 48h.
measure: Pharmacodynamics (IL-1Ra in plasma)
timeFrame: Prior to administration of the IP (Day 0) and repeated assessments during the first 48h.
Other outcomes:
Locations:
city: Copenhagen
country: Denmark
facility: Research Site
Study officials:
affiliation: DanTrials ApS, Copenhagen, Denmark
name: Jesper Sonne, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: AstraZeneca R&D, Lund, Sweden
name: Sam Lindgren, MD, PhD
role: STUDY_DIRECTOR
References: