Clinical trial NCT02944487: A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects
Official title: A Randomised, Double-blind, Placebo-controlled Ascending Dose Tolerance Study of OGT 923 in Healthy Male Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy Subjects
Keywords: safety
pharmacokinetics
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Sponsor class: INDUSTRY
First posted: 2016-10-26
Enrollment: 39
Sex: MALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: The objectives of this study were to evaluate the safety and tolerability of lucerastat, and to determine its pharmacokinetic profile as single oral doses at different strengths.

Detailed description: The subjects were enrolled sequentially to five dose groups, starting with the lowest dose level. Subjects could participate in only one Group.

Eligibility criteria: Inclusion Criteria:

* Signed informed consent form.
* Male subjects aged from 18 to 45 years at screening.
* Body weight between 50 and 100 kg and body mass index (BMI) between 18.0 and 29.0 kg/m2 at screening.
* Healthy on the basis of physical examination, cardiovascular assessments and laboratory tests.

Exclusion Criteria:

* History or clinical evidence of any disease or medical / surgical condition or treatment, which may put the subject at risk of participation in the study or may interfere with the absorption, distribution, metabolism or excretion of the study treatments.
* Serious adverse reaction or hypersensitivity to any drug.
* Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.

Primary outcomes:
measure: Number of participants with Adverse Events (AEs)
timeFrame: From baseline up to 7 days post-administration
measure: Maximum plasma concentration (Cmax) of lucerastat after single ascending doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
measure: Maximum plasma concentration (Cmax) of lucerastat after two daily doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
measure: Time to reach Cmax (tmax) of lucerastat after single ascending doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
measure: Time to reach Cmax (tmax) of lucerastat after two daily doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
measure: Area under the plasma concentration-time curves (AUC) of lucerastat after single ascending doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
measure: Area under the plasma concentration-time curves (AUC) of lucerastat after two daily doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
measure: Terminal elimination half-life (t1/2) of lucerastat after single ascending doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
measure: Terminal elimination half-life (t1/2) of lucerastat after two daily doses of lucerastat
timeFrame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Secondary outcomes:
measure: Change from baseline in heart rate
timeFrame: Up to 24 hours post administration
measure: Change from baseline in blood pressure
timeFrame: Up to 24 hours post administration
measure: Change from baseline in electrocardiogram (ECG) variables
timeFrame: Up to 24 hours post administration
measure: Change from baseline in laboratory tests
timeFrame: Up to 24 hours post administration
Other outcomes:
Locations:
city: Tranent
country: United Kingdom
facility: Investigator Site
Study officials:
affiliation: Idorsia Pharmaceuticals
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Guerard N, Morand O, Dingemanse J. Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects. Orphanet J Rare Dis. 2017 Jan 14;12(1):9. doi: 10.1186/s13023-017-0565-9.