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
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