Gaucher's DiseasePhase 1AcronymNCT06193304
A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
A Phase 1 study testing Eliglustat in people with Gaucher's Disease, run by Sanofi at 1 site in United States.
Trial summary
A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 55 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
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Aug 2014to 21 Sep 2014
- 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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Body weight between 50.0 and 100.0 kg, inclusive, for males, and between 40.0 and 90.0 kg, inclusive, for females, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Having given written informed consent prior to undertaking any study-related procedure.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, and ecg).
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.
- Any subject who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development.
- 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
Covance Clinical Researsh Unit
Evansville, Indiana, 47710
About this study
Official title: An Open-label Study to Assess the Absorption Through the Mouth After Three Repeated 50 mg Doses of Eliglustat Solution, Separated by 2-hour Intervals, Held in the Mouth for 30 Seconds With Swishing But Without Ingestion, in Healthy Subjects
A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects.
Read the detailed description
Duration of the study for each subject, not including screening, will be 3 days including follow-up.
- 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:solution -Route of administration: Oral wihtout ingestion
| Study group | Role | What they receive |
|---|---|---|
| Eliglustat | Experimental | Three repeated doses of eliglustat solution, separated by 2-hour intervals, held in the mouth for 30 seconds with swishing but without ingestion Receives: Drug: Eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma pharmacokinetic (PK) parameter: Cmax
Maximum plasma concentration observed (Cmax)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter tmax
Time to reach Cmax (tmax)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUClast
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast (AUClast)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 0-2h
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to 2 hours (h) post dose (AUC0-2h)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 2-4h
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 2 hours post dose to 4 hours post dose (AUC2-4h)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 4-6h
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 4 hours post dose to 6 hours post dose (AUC4-6h)
Measured: Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter tlast
Measured: Multiple timepoints on Day 1
Secondary outcomes (1)
Treatment emergent adverse events during the study
Measured: Up to Day 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start25 Aug 2014
- Primary completion21 Sep 2014
- Study completion21 Sep 2014
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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 6 participants
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06193304
- Sponsor's protocol ID
- PKM14187
- Other IDs
- U1111-1294-8055
- Acronym
- Acronym
- Status last verified
- Dec 2023
- First submitted
- 21 Dec 2023
- Last updated
- 5 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 5 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study to Assess the Absorption Through the Mouth After Three Repeated 50 mg Doses of Eliglustat Solution, Separated by 2-hour Intervals, Held in the Mouth for 30 Seconds With Swishing But Without Ingestion, in Healthy Subjects
- ClinicalTrials.gov ID
- NCT06193304
- Acronym / sponsor ID
- Acronym / PKM14187
- Conditions
- Gaucher's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 25 Aug 2014 (actual)
- Primary completion
- 21 Sep 2014 (actual)
- Study completion
- 21 Sep 2014 (actual)
- First posted
- 5 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.
A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects.
Detailed description
Duration of the study for each subject, not including screening, will be 3 days including follow-up.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eliglustat | Experimental | Drug: EliglustatThree repeated doses of eliglustat solution, separated by 2-hour intervals, held in the mouth for 30 seconds with swishing but without ingestion |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Body weight between 50.0 and 100.0 kg, inclusive, for males, and between 40.0 and 90.0 kg, inclusive, for females, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Having given written informed consent prior to undertaking any study-related procedure.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, and ecg).
Key exclusion criteria (4 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.
- Any subject who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development.
- The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.
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:solution -Route of administration: Oral wihtout ingestion | Drug | Eliglustat |
Primary outcome
Plasma pharmacokinetic (PK) parameter: Cmax
Time frame: Multiple timepoints on Day 1
Maximum plasma concentration observed (Cmax)
Plasma pharmacokinetic (PK) parameter tmax
Time frame: Multiple timepoints on Day 1
Time to reach Cmax (tmax)
Plasma pharmacokinetic (PK) parameter AUClast
Time frame: Multiple timepoints on Day 1
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast (AUClast)
Plasma pharmacokinetic (PK) parameter AUC 0-2h
Time frame: Multiple timepoints on Day 1
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to 2 hours (h) post dose (AUC0-2h)
Plasma pharmacokinetic (PK) parameter AUC 2-4h
Time frame: Multiple timepoints on Day 1
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 2 hours post dose to 4 hours post dose (AUC2-4h)
Plasma pharmacokinetic (PK) parameter AUC 4-6h
Time frame: Multiple timepoints on Day 1
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 4 hours post dose to 6 hours post dose (AUC4-6h)
Plasma pharmacokinetic (PK) parameter tlast
Time frame: Multiple timepoints on Day 1
1 secondary outcome
Treatment emergent adverse events during the study
Time frame: Up to Day 3
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 5 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 3 inclusion and 4 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 5 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?
Plasma pharmacokinetic (PK) parameter: Cmax (time frame: Multiple timepoints on Day 1). The study lists 7 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 5 Jan 2024.