A Study to Assess the Safety, Tolerability and Pharmacokinetics of Lucerastat (CDP923) After Multiple Dosing in Healthy Subjects

NCT02944474 | Completed Phase 1

• 1 other identifier: CDP923-002
• Study Type: interventional
• Target: 37
• 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 after multiple dosing. Also, the potential effect of food on the pharmacokinetics of lucerastat was explored following a single dose of 500 mg.

Conditions

Healthy Subjects

Keywords

Pharmacokinetics; Safety

Outcome Measures

Primary Outcomes (9)

• Dose proportionality in lucerastat pharmacokinetics assessed by maximum plasma concentration (Cmax)

  Cmax was used to assess dose proportionality across all dose groups

  PK blood samples were collected on Day 7, at pre-dose and at scheduled time points up to 48 hours after the morning dose

• Dose proportionality in lucerastat pharmacokinetics assessed by area under the concentration-time curve (AUC)

  AUC from time zero to infinity \[AUC(0-inf)\] was used to assess dose proportionality across all dose groups

  PK blood samples were collected on Day 7, at pre-dose and at scheduled time points up to 48 hours after the morning dose on Day 7

• Terminal elimination half-life (t1/2)

  t1/2 was calculated from the plasma concentrations-time curves of lucerastat after multiple doses

  PK blood samples were collected on Day 7, at pre-dose and at scheduled time points up to 48 hours after the morning dose on Day 7

• Food effect on lucerastat pharmacokinetics assessed by Cmax

  Potential food effect on pharmacokinetic parameters of lucerastat was tested by comparing Cmax in fed vs fasted state in the 500 mg cohort (cohort 2)

  PK blood samples were collected on Day 1, at pre-dose and at scheduled time points up to 12 hours after the morning dose

• Food effect on lucerastat pharmacokinetics assessed by AUC

  Potential food effect on pharmacokinetic parameters of lucerastat was tested by comparing AUC in fed versus fasted state in the 500 mg cohort (cohort 2)

  PK blood samples were collected on Day 1, at pre-dose and at scheduled time points up to 12 hours after the morning dose

• 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 Day 14 (end of study)

• Change from baseline in haematology after multiple doses of lucerastat

  Up to Day 9

• Change from baseline in clinical chemistry after multiple doses of lucerastat

  Up to Day 9

• Change from baseline in heart rate after multiple doses of lucerastat

  Up to Day 9

Secondary Outcomes (7)

• Change from baseline in haematology after a single dose of lucerastat

  At 24 hours post dose

• Change from baseline in clinical chemistry after a single dose of lucerastat

  At 24 hours post dose

• Change from baseline in heart rate after a single dose of lucerastat

  At 24 hours post dose

• Change from baseline in blood pressure after a single dose of lucerastat

  Up to 24 hours post dose

• Change from baseline in electrocardiogram (ECG) variables after a single dose of lucerastat

  Up to 24 hours post dose

Study Arms (5)

Cohort 1 (200 mg)

EXPERIMENTAL

Six subjects received 200 mg of lucerastat twice daily for 7 consecutive days in fasting conditions

Drug: Lucerastat

Cohort 2 (500 mg)

EXPERIMENTAL

Six subjects received 500 mg of lucerastat as a single dose in the morning of Day 1 in fed conditions. After a 5-day washout, they received the same dose twice daily for 7 consecutive days in fasting conditions

Drug: Lucerastat

Cohort 3 (500 mg)

EXPERIMENTAL

Six subjects received 500 mg of lucerastat twice daily for 7 consecutive days in fasting conditions

Drug: Lucerastat

Cohort 4 (1000 mg)

EXPERIMENTAL

Six subjects received 1 g of lucerastat for 7 consecutive days in fasting conditions

Drug: Lucerastat

Placebo cohorts 1 to 4

PLACEBO COMPARATOR

Twelve subjects received matched placebo (3 subjects per cohort, except for Cohort 3 where 4 subjects received placebo)

Drug: Placebo

Interventions

Lucerastat DRUG

Capsule for oral administration containing lucerastat

Also known as: OGT-923, ACT-434964, CDP923

Cohort 1 (200 mg); Cohort 2 (500 mg); Cohort 3 (500 mg); Cohort 4 (1000 mg)

Placebo DRUG

Placebo capsules matching lucerastat capsules

Placebo cohorts 1 to 4

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

Edinburgh, EH14 4AP, 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 Ltd.

  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: December 1, 2003
• Primary Completion: May 1, 2004
• Study Completion: May 1, 2004
• Last Updated: May 2, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)