A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects

NCT02944487 | Completed Phase 1

• 1 other identifier: OGT923-001
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy Subjects

Keywords

safety; pharmacokinetics

Outcome Measures

Primary Outcomes (9)

• 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

• 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

Secondary Outcomes (4)

• 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

  Up to 24 hours post administration

• Change from baseline in laboratory tests

  Up to 24 hours post administration

Study Arms (4)

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

Drug: Lucerastat

B.i.d. Dose Group

EXPERIMENTAL

Subjects received two doses of lucerastat (2 x 1 g) 12 hours apart on Day 1

Drug: Lucerastat

Placebo for singe ascending doses

PLACEBO COMPARATOR

These subjects received matching placebo administered orally in the morning of Day 1

Drug: Placebo

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

Drug: Placebo

Interventions

Lucerastat DRUG

Hard gelatin capsule for oral administration containing lucerastat

Also known as: OGT-923, ACT-434964

B.i.d. Dose Group; Single ascending doses of lucerastat

Placebo DRUG

Matching placebo capsules

Placebo for b.i.d.Group; Placebo for singe ascending doses

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Idorsia Pharmaceuticals Ltd.: lead

Study Sites (1)

Investigator Site

Tranent, EH33 2NE, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 28088251 DERIVED

MeSH Terms

Interventions

migalastat

Study Officials

• Clinical Trials

  Idorsia Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2016
• First Posted: October 26, 2016
• Study Start: October 1, 2002
• Primary Completion: December 1, 2002
• Study Completion: December 1, 2002
• Last Updated: May 1, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)