Healthy SubjectsPhase 1NCT02944474
A Study to Assess the Safety, Tolerability and Pharmacokinetics of Lucerastat (CDP923) After Multiple Dosing in Healthy Subjects
A Phase 1 study testing Lucerastat in people with Healthy Subjects, run by Idorsia Pharmaceuticals Ltd. at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Healthy Subjects
- What is being tested?
- Lucerastat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Dec 2003to May 2004
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomised Double-blind, Placebo-controlled, Ascending Multiple Dose Phase 1 Study of CDP923 in Healthy Volunteers to Assess Safety, Tolerability, Pharmacokinetics and Food Effect
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.
Read the detailed description
The subjects were to be enrolled sequentially to three dose groups, starting with the lowest dose level. Subjects could participate in only one Group. Progression to an increased dose of lucerastat was permitted only after review of all data from the previous cohort suggested that it was safe to do so.
- Conditions
- Healthy Subjects
- Being tested
- Lucerastat, Placebo
- Keywords
- Pharmacokinetics, Safety
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LucerastatDrug
Also called OGT-923, ACT-434964, CDP923
Capsule for oral administration containing lucerastat
PlaceboDrug
Placebo capsules matching lucerastat capsules
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (200 mg) | Experimental | Six subjects received 200 mg of lucerastat twice daily for 7 consecutive days in fasting conditions Receives: 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 Receives: Drug: Lucerastat |
| Cohort 3 (500 mg) | Experimental | Six subjects received 500 mg of lucerastat twice daily for 7 consecutive days in fasting conditions Receives: Drug: Lucerastat |
| Cohort 4 (1000 mg) | Experimental | Six subjects received 1 g of lucerastat for 7 consecutive days in fasting conditions Receives: 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) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose proportionality in lucerastat pharmacokinetics assessed by maximum plasma concentration (Cmax)
Cmax was used to assess dose proportionality across all dose groups
Measured: 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
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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
Measured: From baseline up to Day 14 (end of study)
Change from baseline in haematology after multiple doses of lucerastat
Measured: Up to Day 9
Change from baseline in clinical chemistry after multiple doses of lucerastat
Measured: Up to Day 9
Change from baseline in heart rate after multiple doses of lucerastat
Measured: Up to Day 9
Secondary outcomes (7)
Change from baseline in haematology after a single dose of lucerastat
Measured: At 24 hours post dose
Change from baseline in clinical chemistry after a single dose of lucerastat
Measured: At 24 hours post dose
Change from baseline in heart rate after a single dose of lucerastat
Measured: At 24 hours post dose
Change from baseline in blood pressure after a single dose of lucerastat
Measured: Up to 24 hours post dose
Change from baseline in electrocardiogram (ECG) variables after a single dose of lucerastat
Measured: Up to 24 hours post dose
Stool frequency after multiple doses of lucerastat
Measured: Every day up to Day 9
Change from baseline in body weight after multiple doses of lucerastat
Measured: At Day 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2016 (estimated)
- Study startDec 2003
- Primary completionMay 2004
- Study completionMay 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Healthy SubjectsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Idorsia Pharmaceuticals Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02944474
- Sponsor's protocol ID
- CDP923-002
- Status last verified
- Apr 2025
- First submitted
- 24 Oct 2016
- Last updated
- 2 May 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 28088251
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised Double-blind, Placebo-controlled, Ascending Multiple Dose Phase 1 Study of CDP923 in Healthy Volunteers to Assess Safety, Tolerability, Pharmacokinetics and Food Effect
- ClinicalTrials.gov ID
- NCT02944474
- Acronym / sponsor ID
- CDP923-002
- Conditions
- Healthy Subjects
- Keywords
- Pharmacokinetics, Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- Dec 2003
- Primary completion
- May 2004 (actual)
- Study completion
- May 2004 (actual)
- First posted
- 26 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The subjects were to be enrolled sequentially to three dose groups, starting with the lowest dose level. Subjects could participate in only one Group. Progression to an increased dose of lucerastat was permitted only after review of all data from the previous cohort suggested that it was safe to do so.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (200 mg) | Experimental | Drug: LucerastatSix subjects received 200 mg of lucerastat twice daily for 7 consecutive days in fasting conditions |
| Cohort 2 (500 mg) | Experimental | Drug: LucerastatSix 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 |
| Cohort 3 (500 mg) | Experimental | Drug: LucerastatSix subjects received 500 mg of lucerastat twice daily for 7 consecutive days in fasting conditions |
| Cohort 4 (1000 mg) | Experimental | Drug: LucerastatSix subjects received 1 g of lucerastat for 7 consecutive days in fasting conditions |
| Placebo cohorts 1 to 4 | Placebo comparator | Drug: PlaceboTwelve subjects received matched placebo (3 subjects per cohort, except for Cohort 3 where 4 subjects received placebo) |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LucerastatAlso: OGT-923, ACT-434964, CDP923Capsule for oral administration containing lucerastat | Drug | Cohort 1 (200 mg), Cohort 2 (500 mg), Cohort 3 (500 mg), Cohort 4 (1000 mg) |
| PlaceboPlacebo capsules matching lucerastat capsules | Drug | Placebo cohorts 1 to 4 |
Primary outcome
Dose proportionality in lucerastat pharmacokinetics assessed by maximum plasma concentration (Cmax)
Time frame: PK blood samples were collected on Day 7, at pre-dose and at scheduled time points up to 48 hours after the morning dose
Cmax was used to assess dose proportionality across all dose groups
Dose proportionality in lucerastat pharmacokinetics assessed by area under the concentration-time curve (AUC)
Time frame: 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
AUC from time zero to infinity [AUC(0-inf)] was used to assess dose proportionality across all dose groups
Terminal elimination half-life (t1/2)
Time frame: 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
t1/2 was calculated from the plasma concentrations-time curves of lucerastat after multiple doses
Food effect on lucerastat pharmacokinetics assessed by Cmax
Time frame: PK blood samples were collected on Day 1, at pre-dose and at scheduled time points up to 12 hours after the morning dose
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)
Food effect on lucerastat pharmacokinetics assessed by AUC
Time frame: PK blood samples were collected on Day 1, at pre-dose and at scheduled time points up to 12 hours after the morning dose
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)
Number of participants with adverse events (AEs)
Time frame: From baseline up to Day 14 (end of study)
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
Change from baseline in haematology after multiple doses of lucerastat
Time frame: Up to Day 9
Change from baseline in clinical chemistry after multiple doses of lucerastat
Time frame: Up to Day 9
Change from baseline in heart rate after multiple doses of lucerastat
Time frame: Up to Day 9
7 secondary outcomes
Change from baseline in haematology after a single dose of lucerastat
Time frame: At 24 hours post dose
Change from baseline in clinical chemistry after a single dose of lucerastat
Time frame: At 24 hours post dose
Change from baseline in heart rate after a single dose of lucerastat
Time frame: At 24 hours post dose
Change from baseline in blood pressure after a single dose of lucerastat
Time frame: Up to 24 hours post dose
Change from baseline in electrocardiogram (ECG) variables after a single dose of lucerastat
Time frame: Up to 24 hours post dose
Stool frequency after multiple doses of lucerastat
Time frame: Every day up to Day 9
Change from baseline in body weight after multiple doses of lucerastat
Time frame: At Day 9
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2025.
- Derived from this study: PubMed 28088251Guerard 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Idorsia Pharmaceuticals Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Lucerastat in people with Healthy Subjects, run by Idorsia Pharmaceuticals Ltd. at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose proportionality in lucerastat pharmacokinetics assessed by maximum plasma concentration (Cmax) (time frame: PK blood samples were collected on Day 7, at pre-dose and at scheduled time points up to 48 hours after the morning dose). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02944474, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2025.