Gaucher's DiseasePhase 1NCT06188325
A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers
A Phase 1 study testing Eliglustat in people with Gaucher's Disease, run by Sanofi at 1 site in United States.
Trial summary
The primary objective of the study is to evaluate dose proportionality and pharmacokinetics for three different dose levels of eliglustat after single and repeated administration. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Gaucher's Disease
- What is being tested?
- Eliglustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2018to 26 Mar 2018
- 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, electrocardiograms (ECG)).
- Having given written informed consent prior to undertaking any study-related procedure
- Having given written informed consent prior to undertaking any study-related procedure
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.
- The following classes of drugs administered within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy:
- Drugs that are strong inducers of CYP3A (eg, rifampin, carbamazepine, phenobarbital,phenytoin, St. John's Wort).
- Drugs that inhibit CYP2D6 or CYP3A (eg, paroxetine, ketoconazole, fluconazole,ranitidine).
- Drugs that are substrates for P-gp (phenytoin, colchicine and dabigatran etexilate) or CYP2D6 (metoprolol, tricyclic antidepressants such as nortriptyline, amitriptyline, or imipramine, and phenothiazines such as perphenazine and chloropromazine).
- The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
M.D.Covance Clinical Research Unit 1341 W
Dallas, Texas, 75247
About this study
Official title: A Randomized, Three-Period Crossover Study of Single and Repeated Doses for Three Different Strengths of Eliglustat in Healthy Adult, CYP2D6 Extensive and Poor Metabolizers
The primary objective of the study is to evaluate dose proportionality and pharmacokinetics for three different dose levels of eliglustat after single and repeated administration.
Read the detailed description
Duration of the study for each subject will be between 42 to 79 days, including a screening period up to 28 days, 3 treatment periods of 7 days each period, a washup period of 7-10 days, and an end-of-study visit 8+/-2 days after the last administration.
- Conditions
- Gaucher's Disease
- Being tested
- Eliglustat
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EliglustatDrug
Also called GZ385660, Cerdelga
Pharmaceutical form:Capsule-Route of administration:Oral
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | CYP2D6 Extensive metabolizers - dose 1, 2 and 3 of eliglustat Receives: Drug: Eliglustat |
| Group 2 | Experimental | CYP2D6 Poor metabolizers - dose 1, 2 and 3 of eliglustat Receives: Drug: Eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic (PK) parameter: Cmax
Eliglustat after single and repeated doses: Maximum plasma concentration observed (Cmax)
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: tmax
Eliglustat after single and repeated doses: Time to reach Cmax
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: AUC0-T
Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve calculated using the trapezoidal method (AUC0-T)
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: AUC
Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve (AUC)
Measured: Multiple timepoints up to Day 35
Secondary outcomes (6)
Pharmacokinetic (PK) parameter: AUClast
Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: tlast
Eliglustat after single and repeated doses: Time corresponding to the last concentration above the limit of quantification, Clast
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: CL/F
Eliglustat after single and repeated doses: Eliglustat after repeated dose: CL/F
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: t1/2z
Eliglustat after single and repeated doses: Terminal half-life associated with the terminal slope (λz)
Measured: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: Ctrough
Eliglustat after single and repeated doses: Plasma concentration observed just before treatment administration during repeated dosing
Measured: Multiple timepoints up to Day 35
Number of participants with treatment emergent adverse events, serious adverse events, and adverse event of special interest
Safety was assessed using clinical laboratory evaluations, ECG parameters, and adverse events spontaneously reported by the subject or observed by the Investigator
Measured: Up to Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2024
- Study start1 Jan 2018
- Primary completion26 Mar 2018
- Study completion26 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher's DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06188325
- Sponsor's protocol ID
- PKM14281
- Other IDs
- U1111-1294-8432
- Status last verified
- Dec 2023
- First submitted
- 18 Dec 2023
- Last updated
- 10 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Three-Period Crossover Study of Single and Repeated Doses for Three Different Strengths of Eliglustat in Healthy Adult, CYP2D6 Extensive and Poor Metabolizers
- ClinicalTrials.gov ID
- NCT06188325
- Acronym / sponsor ID
- PKM14281
- Conditions
- Gaucher's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 26 Mar 2018 (actual)
- Study completion
- 26 Mar 2018 (actual)
- First posted
- 3 Jan 2024 (actual)
- 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 primary objective of the study is to evaluate dose proportionality and pharmacokinetics for three different dose levels of eliglustat after single and repeated administration.
Detailed description
Duration of the study for each subject will be between 42 to 79 days, including a screening period up to 28 days, 3 treatment periods of 7 days each period, a washup period of 7-10 days, and an end-of-study visit 8+/-2 days after the last administration.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: EliglustatCYP2D6 Extensive metabolizers - dose 1, 2 and 3 of eliglustat |
| Group 2 | Experimental | Drug: EliglustatCYP2D6 Poor metabolizers - dose 1, 2 and 3 of eliglustat |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, electrocardiograms (ECG)).
- Having given written informed consent prior to undertaking any study-related procedure
- Having given written informed consent prior to undertaking any study-related procedure
Key exclusion criteria (7 listed)
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.
- The following classes of drugs administered within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy:
- Drugs that are strong inducers of CYP3A (eg, rifampin, carbamazepine, phenobarbital,phenytoin, St. John's Wort).
- Drugs that inhibit CYP2D6 or CYP3A (eg, paroxetine, ketoconazole, fluconazole,ranitidine).
- Drugs that are substrates for P-gp (phenytoin, colchicine and dabigatran etexilate) or CYP2D6 (metoprolol, tricyclic antidepressants such as nortriptyline, amitriptyline, or imipramine, and phenothiazines such as perphenazine and chloropromazine).
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 |
|---|---|---|
| EliglustatAlso: GZ385660, CerdelgaPharmaceutical form:Capsule-Route of administration:Oral | Drug | Group 1, Group 2 |
Primary outcome
Pharmacokinetic (PK) parameter: Cmax
Time frame: Multiple timepoints up to Day 35
Eliglustat after single and repeated doses: Maximum plasma concentration observed (Cmax)
Pharmacokinetic (PK) parameter: tmax
Time frame: Multiple timepoints up to Day 35
Eliglustat after single and repeated doses: Time to reach Cmax
Pharmacokinetic (PK) parameter: AUC0-T
Time frame: Multiple timepoints up to Day 35
Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve calculated using the trapezoidal method (AUC0-T)
Pharmacokinetic (PK) parameter: AUC
Time frame: Multiple timepoints up to Day 35
Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve (AUC)
6 secondary outcomes
Pharmacokinetic (PK) parameter: AUClast
Time frame: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: tlast
Time frame: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: CL/F
Time frame: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: t1/2z
Time frame: Multiple timepoints up to Day 35
Pharmacokinetic (PK) parameter: Ctrough
Time frame: Multiple timepoints up to Day 35
Number of participants with treatment emergent adverse events, serious adverse events, and adverse event of special interest
Time frame: Up to Day 42
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 10 Jan 2024.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Eliglustat in people with Gaucher's Disease, run by Sanofi at 1 site in United States.
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 7 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 10 Jan 2024. 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 States. 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. 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?
Pharmacokinetic (PK) parameter: Cmax (time frame: Multiple timepoints up to Day 35). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Jan 2024.