Gaucher DiseasePhase 1NCT02422654
Taste Evaluation of Different Liquid Formulations With Eliglustat
A Phase 1 study testing eliglustat in people with Gaucher Disease, run by Genzyme, a Sanofi Company at 1 site in United States.
Trial summary
Primary Objective: The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations 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 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
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to May 2015
- 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
- Healthy male and female subjects, 18 to 55 years of age, inclusive.
- Normal smell and taste ability to discriminate odor and flavor differences.
Who cannot join
- Poor metabolizer phenotype status for CYP2D6 (predicted based on genotype).
- Subject who has smoked within 3 months of inclusion.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
Investigational Site Number 840001
Evansville, Indiana, 47710
About this study
Official title: A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects
Primary Objective:
The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations in healthy subjects.
Read the detailed description
The total duration of the study for each subject will be approximately 5 weeks (screening period from Day -28 to Day -2, treatment period of 3 days, and follow-up call on Day 5).
- Conditions
- Gaucher 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
Pharmaceutical form:liquid formulation
Route of administration: oral without ingestion
| Study group | Role | What they receive |
|---|---|---|
| Concentration 1 eliglustat in vehicle A | Experimental | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 1 eliglustat in vehicle B | Experimental | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 1 eliglustat in vehicle C | Experimental | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 1 eliglustat in vehicle D | Experimental | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 1 eliglustat in vehicle E | Experimental | Single dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 2 eliglustat in vehicle A | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 2 eliglustat in vehicle B | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 2 eliglustat in vehicle C | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 2 eliglustat in vehicle D | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 2 eliglustat in vehicle E | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 3 eliglustat in vehicle A | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 3 eliglustat in vehicle B | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 3 eliglustat in vehicle C | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 3 eliglustat in vehicle D | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
| Concentration 3 eliglustat in vehicle E | Experimental | Single dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion Receives: Drug: eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Measured: Each day for 3 days immediately post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Measured: Each day for 3 days 5 minutes post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Measured: Each day for 3 days 15 minutes post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Measured: Each day for 3 days 30 minutes post expectorating the sample
Secondary outcomes (1)
Subject's taste preference questionnaire from most preferred to least preferred of the 5 different liquid formulations
Measured: up to 3 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2015 (estimated)
- Study startApr 2015
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02422654
- Sponsor's protocol ID
- ACC14373
- Other IDs
- U1111-1168-5133
- Status last verified
- May 2015
- First submitted
- 14 Apr 2015
- Last updated
- 27 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects
- ClinicalTrials.gov ID
- NCT02422654
- Acronym / sponsor ID
- ACC14373
- Conditions
- Gaucher Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Apr 2015
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 21 Apr 2015 (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.
Primary Objective:
The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations in healthy subjects.
Detailed description
The total duration of the study for each subject will be approximately 5 weeks (screening period from Day -28 to Day -2, treatment period of 3 days, and follow-up call on Day 5).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 15
| Arm | Type | Receives |
|---|---|---|
| Concentration 1 eliglustat in vehicle A | Experimental | Drug: eliglustatSingle dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle B | Experimental | Drug: eliglustatSingle dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle C | Experimental | Drug: eliglustatSingle dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle D | Experimental | Drug: eliglustatSingle dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 1 eliglustat in vehicle E | Experimental | Drug: eliglustatSingle dose of 5 mL solution held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle A | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle B | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle C | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle D | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 2 eliglustat in vehicle E | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle A | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle B | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle C | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle D | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
| Concentration 3 eliglustat in vehicle E | Experimental | Drug: eliglustatSingle dose of 5 mL liquid formulation held in the mouth for 15 seconds with swishing but with no ingestion |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Healthy male and female subjects, 18 to 55 years of age, inclusive.
- Normal smell and taste ability to discriminate odor and flavor differences.
Key exclusion criteria (3 listed)
- Poor metabolizer phenotype status for CYP2D6 (predicted based on genotype).
- Subject who has smoked within 3 months of inclusion.
- The above information is not intended to contain all considerations relevant to a patient's 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: GZ385660Pharmaceutical form:liquid formulation Route of administration: oral without ingestion | Drug | Concentration 1 eliglustat in vehicle A, Concentration 1 eliglustat in vehicle B, Concentration 1 eliglustat in vehicle C, Concentration 1 eliglustat in vehicle D, Concentration 1 eliglustat in vehicle E, Concentration 2 eliglustat in vehicle A, Concentration 2 eliglustat in vehicle B, Concentration 2 eliglustat in vehicle C, Concentration 2 eliglustat in vehicle D, Concentration 2 eliglustat in vehicle E, Concentration 3 eliglustat in vehicle A, Concentration 3 eliglustat in vehicle B, Concentration 3 eliglustat in vehicle C, Concentration 3 eliglustat in vehicle D, Concentration 3 eliglustat in vehicle E |
Primary outcome
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Time frame: Each day for 3 days immediately post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Time frame: Each day for 3 days 5 minutes post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Time frame: Each day for 3 days 15 minutes post expectorating the sample
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability
Time frame: Each day for 3 days 30 minutes post expectorating the sample
1 secondary outcome
Subject's taste preference questionnaire from most preferred to least preferred of the 5 different liquid formulations
Time frame: up to 3 days
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 27 May 2015.
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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 Sciences & Operations study director
Sanofi
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 Disease, run by Genzyme, a Sanofi Company 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 2 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 27 May 2015. 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?
Measurement of palatability by 100 mm visual analogue scale (VAS) for overall acceptability (time frame: Each day for 3 days immediately post expectorating the sample). 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 27 May 2015.