Gaucher DiseasePhase 1NCT02536937
A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
A Phase 1 study testing eliglustat in people with Gaucher Disease, run by Sanofi at 3 sites in United States.
Trial summary
Primary Objective: To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild, moderate, and severe renal impairment in comparison with matched… Read more about this study
- Who is it for?
- Ages 18 to 79 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
- 32 participants
- Where
- 3 sitesUnited States
- Study dates
- Sep 2015to Jan 2017
- 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 79 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- For renal impaired:
- Male or female subjects, between 18 and 79 years of age, inclusive.
- Body weight between 50.0 kg and 125.0 kg inclusive if male, between 40.0 kg and 110.0 kg inclusive if female, body mass index (BMI) between 18.0 and 37.0 kg/m^2, inclusive.
- Stable chronic renal impairment, as defined by Cockroft-Gault formula.
- For severe renal impairment: CrCl <30 mL/min.
- For moderate renal impairment: 30 mL/min ≤CrCl <50 mL/min.
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- For mild renal impairment: 50 mL/min ≤CrCl ≤80 mL/min.
- For matched subjects:
- Male or female subject, between 18 and 79 years inclusive, matched by age.
- Body weight within 15% of the body weight of the subjects with renal impairment to be matched and BMI between 18.0 and 37.0 mg/kg^2 inclusive.
- Matched by cytochrome P450 (CYP) 2D6 predicted phenotype based on genotype.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical examination).
- For healthy subjects: CrCl >80 mL/min.
Who cannot join
- For renal impairment patients:
- Uncontrolled clinically relevant cardiovascular, pulmonary, gastrointestinal, metabolic, hematological, neurological, psychiatric, systemic, ocular, gynecologic (if female) or infectious disease, or signs of acute illness.
- Active hepatitis, hepatic insufficiency.
- Acute renal failure (de novo or superimposed to pre-existing chronic renal impairment), nephrotic syndrome.
- History of or current hematuria of urologic origin that limits the subject's participation in the study.
- Subjects requiring dialysis during the study.
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- Presence or history of drug hypersensitivity, or allergic disease diagnosed and treated by a physician.
- If female, pregnancy (defined as positive beta-human chorionic gonadotropin [β-hCG] blood test), breastfeeding.
- Any significant change in chronic treatment medication within 14 days before inclusion.
- P-gp inhibitors and/or inducers, CYP2D6 and/or CYP3A inducers, and strong and/or moderate CYP2D6 and/or CYP3A inhibitors. Up to one weak CYP2D6 inhibitor and/or one weak CYP3A inhibitor are allowed (as defined in The Metabolism and Transport Drug Interaction Database™ (DIDB).
- Positive result on any of the following tests: anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 Ab).
- Pre-existing cardiac disease (current congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or use of Class IA (eg, quinidine, procainamide) and Class III (eg, amiodarone, sotalol) anti-arrhythmic medications.
- Any subject with CYP2D6 indeterminate or ultra-rapid metabolizer (URM) phenotype.
- For matched volunteers:
- 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.
- Presence or history of drug hypersensitivity, or allergic disease diagnosed and treated by a physician.
- If female, pregnancy (defined as positive β-hCG blood test), breast feeding.
- For subjects 50 years old and below: any medication (including St John's Wort) within 14 days before inclusion, or within 5 times the elimination half-life or pharmacodynamic half-life of that medication, whichever is longest, with the exception of hormonal contraception or menopausal hormone replacement therapy; any vaccination within the last 28 days, and any biologics (antibody or its derivatives) within 4 months before inclusion.
- For subjects above 50 years old: any significant change in chronic treatment medication within 14 days before inclusion.
- P-gp inhibitors and/or inducers, CYP2D6 and/or CYP3A inducers, and strong and/or moderate CYP2D6 and/or CYP3A inhibitors. Up to one weak CYP2D6 inhibitor and/or one weak CYP3A inhibitor are allowed (as defined in The Metabolism and Transport Drug Interaction Database™ (DIDB).
- Positive result on any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) Ab, anti-HIV1 and anti-HIV2 Ab.
- Pre-existing cardiac disease (current congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or use of Class IA (eg, quinidine, procainamide) and Class III (eg, amiodarone, sotalol) anti-arrhythmic medications.
- 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
3 sites in 1 country.
United States3 sites
Investigational Site Number 840001
Knoxville, Tennessee, 37920
Investigational Site Number 840004
Miami, Florida, 33014
Investigational Site Number 840002
Saint Paul, Minnesota, 55144
About this study
Official title: An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function
Primary Objective:
To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat.
Secondary Objective:
To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild, moderate, and severe renal impairment in comparison with matched subjects with normal renal function.
Read the detailed description
The total study duration from screening period is approximately 31 days. In stage 1, only subjects with severe renal impairment and normal renal function will be enrolled. Subjects with mild and moderate renal impairment may be enrolled in stage 2 if the results in subjects with severe renal impairment show a substantial effect of reduced renal function on pharmacokinetics.
- 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: capsule
Route of administration: oral
| Study group | Role | What they receive |
|---|---|---|
| GZ385660 (healthy subjects) | Experimental | Single dose of eliglustat tartrate will be given under fed conditions Receives: Drug: eliglustat |
| GZ385660 (subjects with mild renal impairment) | Experimental | Single dose of eliglustat tartrate will be given under fed conditions Receives: Drug: eliglustat |
| GZ385660 (subjects with moderate renal impairment) | Experimental | Single dose of eliglustat tartrate will be given under fed conditions Receives: Drug: eliglustat |
| GZ385660 (subjects with severe renal impairment) | Experimental | Single dose of eliglustat tartrate will be given under fed conditions Receives: Drug: eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
- Assessment of PK parameter: Maximum plasma concentration observed (Cmax)
Measured: 3 days
- Assessment of PK parameter: Area under the plasma concentration (AUC)
Measured: 3 days
Secondary outcomes (6)
Assessment of PK parameter: Area under the plasma concentration versus time curve (AUClast)
Measured: 3 days
Assessment of PK parameter: Apparent total body clearance (CL/F)
Measured: 3 days
Assessment of PK parameter: Apparent volume of distribution during the terminal phase (Vz/F)
Measured: 3 days
Assessment of PK parameter: Predicted accumulation ratio (Rac,pred)
Measured: 3 days
Assessment of PK parameter: Terminal half-life (t1/2z)
Measured: 3 days
Number of adverse events
Measured: Up to 10 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2015 (estimated)
- Study startSep 2015
- Primary completionJan 2017
- Study completionJan 2017
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.
- 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02536937
- Sponsor's protocol ID
- POP13778
- Other IDs
- U1111-1170-3686
- Status last verified
- Mar 2017
- First submitted
- 28 Aug 2015
- Last updated
- 8 Mar 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function
- ClinicalTrials.gov ID
- NCT02536937
- Acronym / sponsor ID
- POP13778
- Conditions
- Gaucher Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- Sep 2015
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 1 Sep 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:
To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat.
Secondary Objective:
To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild, moderate, and severe renal impairment in comparison with matched subjects with normal renal function.
Detailed description
The total study duration from screening period is approximately 31 days. In stage 1, only subjects with severe renal impairment and normal renal function will be enrolled. Subjects with mild and moderate renal impairment may be enrolled in stage 2 if the results in subjects with severe renal impairment show a substantial effect of reduced renal function on pharmacokinetics.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| GZ385660 (healthy subjects) | Experimental | Drug: eliglustatSingle dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with mild renal impairment) | Experimental | Drug: eliglustatSingle dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with moderate renal impairment) | Experimental | Drug: eliglustatSingle dose of eliglustat tartrate will be given under fed conditions |
| GZ385660 (subjects with severe renal impairment) | Experimental | Drug: eliglustatSingle dose of eliglustat tartrate will be given under fed conditions |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Male or female subjects, between 18 and 79 years of age, inclusive.
- Body weight between 50.0 kg and 125.0 kg inclusive if male, between 40.0 kg and 110.0 kg inclusive if female, body mass index (BMI) between 18.0 and 37.0 kg/m^2, inclusive.
- Stable chronic renal impairment, as defined by Cockroft-Gault formula.
- For severe renal impairment: CrCl <30 mL/min.
- For moderate renal impairment: 30 mL/min ≤CrCl <50 mL/min.
Key exclusion criteria (23 listed)
- Uncontrolled clinically relevant cardiovascular, pulmonary, gastrointestinal, metabolic, hematological, neurological, psychiatric, systemic, ocular, gynecologic (if female) or infectious disease, or signs of acute illness.
- Active hepatitis, hepatic insufficiency.
- Acute renal failure (de novo or superimposed to pre-existing chronic renal impairment), nephrotic syndrome.
- History of or current hematuria of urologic origin that limits the subject's participation in the study.
- Subjects requiring dialysis during the study.
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: capsule Route of administration: oral | Drug | GZ385660 (healthy subjects), GZ385660 (subjects with mild renal impairment), GZ385660 (subjects with moderate renal impairment), GZ385660 (subjects with severe renal impairment) |
Primary outcome
- Assessment of PK parameter: Maximum plasma concentration observed (Cmax)
Time frame: 3 days
- Assessment of PK parameter: Area under the plasma concentration (AUC)
Time frame: 3 days
6 secondary outcomes
Assessment of PK parameter: Area under the plasma concentration versus time curve (AUClast)
Time frame: 3 days
Assessment of PK parameter: Apparent total body clearance (CL/F)
Time frame: 3 days
Assessment of PK parameter: Apparent volume of distribution during the terminal phase (Vz/F)
Time frame: 3 days
Assessment of PK parameter: Predicted accumulation ratio (Rac,pred)
Time frame: 3 days
Assessment of PK parameter: Terminal half-life (t1/2z)
Time frame: 3 days
Number of adverse events
Time frame: Up to 10 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 8 Mar 2017.
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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
3 sites 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 Sanofi at 3 sites 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 13 inclusion and 23 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 8 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
- Assessment of PK parameter: Maximum plasma concentration observed (Cmax) (time frame: 3 days). The study lists 2 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 8 Mar 2017.