Healthy VolunteersPhase 1NCT03687554
Effect of Venglustat in Patients With Renal Impairment
A Phase 1 study testing Venglustat GZ/SAR402671 in people with Healthy Volunteers and Polycystic Kidney, Autosomal Dominant, run by Genzyme, a Sanofi Company at 1 site in United States.
Trial summary
Primary Objective: To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose. Secondary Objective: To assess the tolerability of Venglustat given as a single dose in subjects with mild, moderate and severe renal impairment in comparison with… Read more about this study
- Who is it for?
- Ages 18 to 79 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
- What is being tested?
- Venglustat GZ/SAR402671
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Oct 2018to 27 Feb 2019
- 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 all Subjects:
- Male and/or female subjects, between 18 and 79 years of age, inclusive.
- Body weight between 50.0 and 115.0 kg, inclusive, if male, and between 40.0 and 100.0 kg, inclusive, if female, body mass index between 18.0 and 34.9 kg/m2, inclusive
- Normal electrocardiogram (ECG)
- Having given written informed consent prior to undertaking any study-related procedure
- Not under any administrative or legal supervision
Show all 17Show fewer
- Male subject, whose partners are of childbearing potential (including lactating women), must accept to use, during sexual intercourse, a double contraception method according to the following algorithm: (condom) plus (spermicide or intra-uterine device or hormonal contraceptive) from the inclusion up to 4 months after the last dosing
- Male subject, whose partners are pregnant, must use, during sexual intercourse, a condom from the inclusion up to 4 months after the last dosing
- Male subject has agreed not to donate sperm from the inclusion up to 4 months after the last dosing
- Female subject must use a double contraception method including a highly effective method of birth control from at least 30 days prior to the inclusion to 30 days after the last IMP administration, except if she has undergone sterilization (documented) at least 3 months earlier or is postmenopausal
- Specific for subjects with renal impairment:
- Stable chronic renal impairment
- Vital signs and laboratory parameters within acceptable range for subjects with renal impairment
- Specific for matched healthy subjects:
- Normal renal function
- Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical exam)
- Normal vital signs and laboratory parameters
Who cannot join
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month)
- Blood donation, any volume, within 2 months before inclusion
- Symptomatic postural hypotension, irrespective of the decrease in blood pressure, or asymptomatic postural hypotension judged clinically relevant by the Investigator
- Any significant change in chronic treatment medication within 14 days before inclusion
- Any drug which could impact by any mechanism of action, the pharmacokinetics of the investigational medicinal product, including moderate and strong cytochrome P3A (CYP3A) inhibitors or inducers; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion
- Positive result on any of the following tests: hepatitis B surface (HBs Ag) antigen, anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti HIV2 Ab)
Show all 17Show fewer
- Positive result on urine drug screen or plasma alcohol test
- Active hepatitis, hepatic insufficiency
- If female, pregnancy [defined as positive β-Human Chorionic Gonadotropin (β-HCG) blood test], breast-feeding
- Specific for subjects with renal impairment:
- Uncontrolled clinically relevant cardiovascular, pulmonary, gastrointestinal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness
- Acute renal failure (de novo or superimposed on preexisting 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
- Specific for matched healthy controls:
- 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 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 8400001
Miami, Florida, 33014
About this study
Official title: A Phase I, Single-Center, Open-label, Single Dose Pharmacokinetic and Tolerability Study of GZ402671 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 Venglustat following a single dose.
Secondary Objective:
To assess the tolerability of Venglustat 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
Approximately 41 days, including a 21-day screening period, a 1-day treatment period, followed by a 9-day period of plasma sampling for assessment of primary endpoints.
- Being tested
- Venglustat GZ/SAR402671
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venglustat GZ/SAR402671Drug
Pharmaceutical form: Hard Capsule Route of administration: Oral
| Study group | Role | What they receive |
|---|---|---|
| Venglustat | Experimental | Single dose of Venglustat is given, orally under fasting conditions Receives: Drug: Venglustat GZ/SAR402671 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of pharmacokinetic (PK) parameters of Venglustat: Area under the curve (AUC)
Venglustat area under the plasma concentration versus time curve (AUC)
Measured: Day 1 to Day 10
Secondary outcomes (13)
Venglustat plasma pharmacokinetic (PK) parameter: Cmax
Maximum plasma concentration observed (Cmax)
Measured: Day 1
Venglustat plasma pharmacokinetic (PK) parameter: AUClast
Area under the plasma concentration versus time curve calculated from time zero to the real time tlast (AUClast)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: unbound Cmax
Maximum plasma concentration observed of unbound drug (unbound Cmax)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: unbound AUC
Change in unbound Venglustat area under the plasma concentration versus time curve (unbound AUC)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: CL/F
Apparent total body clearance of Venglustat from plasma (CL/F)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: Vss/F
Apparent volume of distribution of Venglustat at steady state (Vss/F)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: fu
Fraction of unbound venglustat in plasma (fu)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: t1/2z
Terminal half-life associated with the terminal slope (t1/2z)
Measured: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: t1/2eff
Effective half-life (t1/2eff)
Measured: Day 1 to Day 10
Venglustat urine pharmacokinetic (PK) parameter: Ae(0-24)
Cumulated amount excreted in urine from time 0 to time 24h after Venglustat administration
Measured: Day 1 and Day 2
Venglustat urine pharmacokinetic (PK) parameter: fe(0-24)
Fraction of dose excreted in urine from time 0 to time 24h after Venglustat administration
Measured: Day 1 and Day 2
Venglustat urine pharmacokinetic (PK) parameter: CLR(0-24)
Renal clearance of the drug determined in the 0-24h interval (CLR(0-24))
Measured: Day 1 and Day 2
Venglustat plasma pharmacokinetic (PK) parameter: Rac,pred
Predicted accumulation ratio (Rac,pred)
Measured: Day 1 to Day 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2018
- Study start5 Oct 2018
- Primary completion27 Feb 2019
- Study completion27 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteersStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 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
- NCT03687554
- Sponsor's protocol ID
- POP14499
- Other IDs
- U1111-1205-3215
- Status last verified
- Apr 2022
- First submitted
- 26 Sep 2018
- Last updated
- 25 Apr 2022
- 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 25 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Single-Center, Open-label, Single Dose Pharmacokinetic and Tolerability Study of GZ402671 in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function
- ClinicalTrials.gov ID
- NCT03687554
- Acronym / sponsor ID
- POP14499
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 5 Oct 2018 (actual)
- Primary completion
- 27 Feb 2019 (actual)
- Study completion
- 27 Feb 2019 (actual)
- First posted
- 27 Sep 2018 (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.
Primary Objective:
To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose.
Secondary Objective:
To assess the tolerability of Venglustat 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
Approximately 41 days, including a 21-day screening period, a 1-day treatment period, followed by a 9-day period of plasma sampling for assessment of primary endpoints.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venglustat | Experimental | Drug: Venglustat GZ/SAR402671Single dose of Venglustat is given, orally under fasting conditions |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male and/or female subjects, between 18 and 79 years of age, inclusive.
- Body weight between 50.0 and 115.0 kg, inclusive, if male, and between 40.0 and 100.0 kg, inclusive, if female, body mass index between 18.0 and 34.9 kg/m2, inclusive
- Normal electrocardiogram (ECG)
- Having given written informed consent prior to undertaking any study-related procedure
- Not under any administrative or legal supervision
Key exclusion criteria (17 listed)
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month)
- Blood donation, any volume, within 2 months before inclusion
- Symptomatic postural hypotension, irrespective of the decrease in blood pressure, or asymptomatic postural hypotension judged clinically relevant by the Investigator
- Any significant change in chronic treatment medication within 14 days before inclusion
- Any drug which could impact by any mechanism of action, the pharmacokinetics of the investigational medicinal product, including moderate and strong cytochrome P3A (CYP3A) inhibitors or inducers; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion
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 |
|---|---|---|
| Venglustat GZ/SAR402671Pharmaceutical form: Hard Capsule Route of administration: Oral | Drug | Venglustat |
Primary outcome
Assessment of pharmacokinetic (PK) parameters of Venglustat: Area under the curve (AUC)
Time frame: Day 1 to Day 10
Venglustat area under the plasma concentration versus time curve (AUC)
13 secondary outcomes
Venglustat plasma pharmacokinetic (PK) parameter: Cmax
Time frame: Day 1
Venglustat plasma pharmacokinetic (PK) parameter: AUClast
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: unbound Cmax
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: unbound AUC
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: CL/F
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: Vss/F
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: fu
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: t1/2z
Time frame: Day 1 to Day 10
Venglustat plasma pharmacokinetic (PK) parameter: t1/2eff
Time frame: Day 1 to Day 10
Venglustat urine pharmacokinetic (PK) parameter: Ae(0-24)
Time frame: Day 1 and Day 2
Venglustat urine pharmacokinetic (PK) parameter: fe(0-24)
Time frame: Day 1 and Day 2
Venglustat urine pharmacokinetic (PK) parameter: CLR(0-24)
Time frame: Day 1 and Day 2
Venglustat plasma pharmacokinetic (PK) parameter: Rac,pred
Time frame: Day 1 to Day 10
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 25 Apr 2022.
- 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
Related trials for Healthy Volunteers
Not yet recruiting
Neuroergonomic Analysis of a Brain-Computer Interface Device (BCI)
Healthy Volunteers - Male and Female
RecruitingPhase 1
First-in-Human Trial to Assess REGN20423 in Healthy Adult Participants and Adult Participants With Atopic Dermatitis
Healthy Volunteer, Atopic Dermatitis
Not yet recruitingObservational study
Establishment of Biobank of Nasal Epithelium Samples From Healthy Volunteers
Healthy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
Not yet recruitingObservational study
Screening Protocol
Pain
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 Venglustat GZ/SAR402671 in people with Healthy Volunteers and Polycystic Kidney, Autosomal Dominant, 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 17 inclusion and 17 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 25 Apr 2022. 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?
Assessment of pharmacokinetic (PK) parameters of Venglustat: Area under the curve (AUC) (time frame: Day 1 to Day 10). 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 25 Apr 2022.