Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
The subjects were enrolled sequentially to five dose groups, starting with the lowest dose level. Subjects could participate in only one Group.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single ascending doses of lucerastat | Experimental | Subjects were enrolled sequentially in 4 groups and received a single oral dose of lucerastat from 100 mg to 1000 mg in the morning of Day 1 |
|
| B.i.d. Dose Group | Experimental | Subjects received two doses of lucerastat (2 x 1 g) 12 hours apart on Day 1 |
|
| Placebo for singe ascending doses | Placebo Comparator | These subjects received matching placebo administered orally in the morning of Day 1 |
|
| Placebo for b.i.d.Group | Placebo Comparator | These subjects received matching placebo administered orally in the morning and in the evening of Day 1 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lucerastat | Drug | Hard gelatin capsule for oral administration containing lucerastat |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with Adverse Events (AEs) | An AE was defined as any untoward medical occurrence in a clinical investigation subject, which did not necessarily have a causal relationship with the treatment. | From baseline up to 7 days post-administration |
| Maximum plasma concentration (Cmax) of lucerastat after single ascending doses of lucerastat | Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose. | PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration |
| Maximum plasma concentration (Cmax) of lucerastat after two daily doses of lucerastat | Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily. | PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration |
| Time to reach Cmax (tmax) of lucerastat after single ascending doses of lucerastat | tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose. | PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration |
| Time to reach Cmax (tmax) of lucerastat after two daily doses of lucerastat | tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily. | PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in heart rate | Up to 24 hours post administration | |
| Change from baseline in blood pressure | Up to 24 hours post administration | |
| Change from baseline in electrocardiogram (ECG) variables |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Idorsia Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site | Tranent | EH33 2NE | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28088251 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C090092 | migalastat |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Matching placebo capsules |
|
| Area under the plasma concentration-time curves (AUC) of lucerastat after single ascending doses of lucerastat |
AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving a single dose. |
| PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration |
| Area under the plasma concentration-time curves (AUC) of lucerastat after two daily doses of lucerastat | AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving lucerastat twice daily | PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration |
| Terminal elimination half-life (t1/2) of lucerastat after single ascending doses of lucerastat | t1/2 was calculated from the corresponding plasma concentrations-time curves for subjects receiving a single dose | PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration |
| Terminal elimination half-life (t1/2) of lucerastat after two daily doses of lucerastat | t1/2 was calculated from the plasma concentrations-time curves for subjects receiving lucerastat twice daily | PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration |
| Up to 24 hours post administration |
| Change from baseline in laboratory tests | Up to 24 hours post administration |