Disorders of Sphingolipid MetabolismPhase 1NCT05238714
Excretion Balance, Pharmacokinetics, and Metabolism Following of [14C]-Venglustat Administration in Healthy Male Subjects
A Phase 1 study testing venglustat in people with Disorders of Sphingolipid Metabolism and Healthy Volunteers, run by Genzyme, a Sanofi Company at 1 site in United Kingdom.
Trial summary
Primary Objectives: To determine the excretion balance and systemic exposure of radioactivity after oral administration of [14C]-venglustat. To determine the pharmacokinetics (PK) of venglustat and its contribution to the overall exposure of radioactivity. Read more about this study
- Who is it for?
- Ages 30 to 65 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Disorders of Sphingolipid Metabolism, Healthy Volunteers
- What is being tested?
- venglustat
- 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 Kingdom
- Study dates
- 26 May 2020to 26 Jun 2020
- 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 30 to 65 years
- Men only
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Body weight between 50.0 and 100.0 kg, inclusive, body Mass Index 18 to 32 kg/m2, inclusive
- Certified as healthy by a comprehensive clinical assessment
- Normal vital signs after 10 minutes resting in supine position
- Standard 12-lead electrocardiogram (ECG) parameters after 10 minutes resting in supine position in the following ranges; 120 ms≤PR≤230 msec, QRS≤120 msec, QTc≤450 msec and normal ECG tracing unless the Investigator considers an ECG tracing abnormality to be not clinically relevant
- Laboratory parameters within the normal range
- Having given written informed consent prior to undertaking any study-related procedure
Show all 10Show fewer
- Covered by a health insurance system where applicable, and/or in compliance with the recommendations of the national laws in force relating to biomedical research
- Not under any administrative or legal supervision
- Normal renal function as expressed by a creatinine clearance >80 mL/min as calculated by the Cockroft and Gault formula
- Male subjects who agree to use condoms, whose female partner(s) are of childbearing potential (including lactating women), must accept to use, during sexual intercourse, a double contraception method (unless they underwent surgical sterilization) until 90 days after the end of study
Who cannot join
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month).
- Blood donation, any volume, within 3 months before inclusion.
- Symptomatic postural hypotension, irrespective of the decrease in blood pressure.
- Presence or history of clinically significant drug hypersensitivity, or allergic disease diagnosed and treated by a physician that in the opinion of the Investigator would compromise subject safety.
- History or presence of drug or alcohol abuse (alcohol consumption more than 14 units per week on a regular basis) in the 5 years prior to screening.
Show all 25Show fewer
- Smoking or using nicotine replacement products or e-cigarettes regularly more than 5 cigarettes or equivalent per week, unable to stop smoking during the study (occasional smoker can be enrolled).
- Excessive consumption of beverages containing xanthine bases (more than 4 cups or glasses per day)
- 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 the medication; any vaccination or any medications known to be CYP3A4 inducers within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion
- Subjects who do not have suitable veins for multiple venepunctures/cannulation as assessed by the Investigator at screening.
- 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.
- Any subject enrolled in or having participated, in [this or] any other clinical study involving an investigational medicinal product (IMP) according to applicable regulations/guidelines in the 3 months prior to dosing of this study.
- Any subject who cannot be contacted in case of emergency.
- Any subject who is the Investigator or any sub investigator, research assistant, pharmacist, study coordinator, or other staff thereof, directly involved in conducting the study
- 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).
- Confirmed positive result on urine drug screen (amphetamines/methamphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine, tricyclic antidepressants, opiates including methadone).
- Positive alcohol breath test
- Any subject with specific dietary habits, such as vegan.
- Any subject with irregular bowel habits (more than 3 bowel movements/day or less than 1 every 2 days).
- Any subject undergoing dental care or presenting with dental caries.
- Any subject who is occupationally exposed to radiation as defined in the Ionizing Radiations Regulations 2017
- Participation in a trial with 14C-radiolabelled medication in the 12 months preceding the study.
- Radiation exposure, including that from the present study and radiopharmaceuticals or radionuclides in therapeutic or diagnostic procedures, but excluding background radiation, exceeding 5 millisievert (mSv) in the last 12 months or 10 mSv in the last 5 years.
- Any consumption of citrus (grapefruit, orange, etc) or their juices within 5 days before inclusion
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Investigational Site Number :8260001
Nottingham, Nottinghamshire, NG11 6JS
About this study
Official title: An Open-label Study of Excretion Balance and Pharmacokinetics Following a Single Oral Dose of [14C]-Venglustat (2.67 MBq) in Healthy Male Subjects
Primary Objectives:
- To determine the excretion balance and systemic exposure of radioactivity after oral administration of [14C]-venglustat.
- To determine the pharmacokinetics (PK) of venglustat and its contribution to the overall exposure of radioactivity.
- To determine the metabolic pathways, metabolite profile, chemical structures and main excretion route of the main venglustat metabolites and the metabolite contribution to the overall exposure of radioactivity.
Secondary Objective:
To assess the clinical and biological tolerability of oral solution of venglustat
Read the detailed description
43 days
- Being tested
- venglustat
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
venglustatDrug
Powder for Oral solution
| Study group | Role | What they receive |
|---|---|---|
| [14C] venglustat | Experimental | Single dose of [14C] venglustat Oral Solution under fasting conditions Receives: Drug: venglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of radioactive dose excreted in urine and faeces
Percentage of radioactive dose excreted in urine and faeces
Measured: Day -1 up to max Day 43
Cmax of plasma and blood radioactivity
Maximum plasma or blood concentration observed
Measured: Day 1 up to max Day 43
AUC Last of plasma and blood radioactivity
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time, tlast (time corresponding to the last concentration above the limit of quantification, Clast)
Measured: Day 1 up to max Day 43
AUC Last of plasma venglustat
Measured: Day 1 up to max Day 43
tmax of plasma and blood radioactivity
Time to Cmax
Measured: Day 1 up to max Day 43
tmax of plasma venglustat
Time to reach Cmax
Measured: Day 1 up to max Day 43
Secondary outcomes (4)
Number of participants with adverse events (AEs)
Number of participants with AEs
Measured: Day 1 up to max Day 43
Percentage of venglustat metabolites in plasma
Percentage of venglustat metabolites in plasma
Measured: Day 1 up to max Day 43
Percentage of venglustat metabolites in urine
Percentage of venglustat metabolites in urine
Measured: Day 1 up to max Day 43
Percentage of venglustat metabolites in faeces
Percentage of venglustat metabolites in faeces
Measured: Day 1 up to max Day 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2022
- Study start26 May 2020
- Primary completion26 Jun 2020
- Study completion26 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Disorders of Sphingolipid MetabolismStudies 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
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 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
- NCT05238714
- Sponsor's protocol ID
- BEX13673
- Other IDs
- 2019-001041-41, U1111-1180-5557, 2019
- Status last verified
- Sep 2025
- First submitted
- 3 Feb 2022
- Last updated
- 17 Sep 2025
- 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 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of Excretion Balance and Pharmacokinetics Following a Single Oral Dose of [14C]-Venglustat (2.67 MBq) in Healthy Male Subjects
- ClinicalTrials.gov ID
- NCT05238714
- Acronym / sponsor ID
- BEX13673
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 26 May 2020 (actual)
- Primary completion
- 26 Jun 2020 (actual)
- Study completion
- 26 Jun 2020 (actual)
- First posted
- 14 Feb 2022 (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 Objectives:
- To determine the excretion balance and systemic exposure of radioactivity after oral administration of [14C]-venglustat.
- To determine the pharmacokinetics (PK) of venglustat and its contribution to the overall exposure of radioactivity.
- To determine the metabolic pathways, metabolite profile, chemical structures and main excretion route of the main venglustat metabolites and the metabolite contribution to the overall exposure of radioactivity.
Secondary Objective:
To assess the clinical and biological tolerability of oral solution of venglustat
Detailed description
43 days
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| [14C] venglustat | Experimental | Drug: venglustatSingle dose of [14C] venglustat Oral Solution under fasting conditions |
Who may qualify
- Ages
- 30 to 65 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Body weight between 50.0 and 100.0 kg, inclusive, body Mass Index 18 to 32 kg/m2, inclusive
- Certified as healthy by a comprehensive clinical assessment
- Normal vital signs after 10 minutes resting in supine position
- Standard 12-lead electrocardiogram (ECG) parameters after 10 minutes resting in supine position in the following ranges; 120 ms≤PR≤230 msec, QRS≤120 msec, QTc≤450 msec and normal ECG tracing unless the Investigator considers an ECG tracing abnormality to be not clinically relevant
- Laboratory parameters within the normal range
Key exclusion criteria (25 listed)
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month).
- Blood donation, any volume, within 3 months before inclusion.
- Symptomatic postural hypotension, irrespective of the decrease in blood pressure.
- Presence or history of clinically significant drug hypersensitivity, or allergic disease diagnosed and treated by a physician that in the opinion of the Investigator would compromise subject safety.
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 |
|---|---|---|
| venglustatPowder for Oral solution | Drug | [14C] venglustat |
Primary outcome
Percentage of radioactive dose excreted in urine and faeces
Time frame: Day -1 up to max Day 43
Percentage of radioactive dose excreted in urine and faeces
Cmax of plasma and blood radioactivity
Time frame: Day 1 up to max Day 43
Maximum plasma or blood concentration observed
AUC Last of plasma and blood radioactivity
Time frame: Day 1 up to max Day 43
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time, tlast (time corresponding to the last concentration above the limit of quantification, Clast)
AUC Last of plasma venglustat
Time frame: Day 1 up to max Day 43
tmax of plasma and blood radioactivity
Time frame: Day 1 up to max Day 43
Time to Cmax
tmax of plasma venglustat
Time frame: Day 1 up to max Day 43
Time to reach Cmax
4 secondary outcomes
Number of participants with adverse events (AEs)
Time frame: Day 1 up to max Day 43
Percentage of venglustat metabolites in plasma
Time frame: Day 1 up to max Day 43
Percentage of venglustat metabolites in urine
Time frame: Day 1 up to max Day 43
Percentage of venglustat metabolites in faeces
Time frame: Day 1 up to max Day 43
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 17 Sep 2025.
- BEX13673 Plain Language Results SummaryLink provided by the sponsor.
- 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 Disorders of Sphingolipid Metabolism
RecruitingObservational study
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Not yet recruitingPhase 2
Research of Traditional Chinese Medicine Oral Preparation of C. Cicadae in the Treatment of ALS Patients With Elevated Plasma Sphingolipids
Amyotrophic Lateral Sclerosis
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 in people with Disorders of Sphingolipid Metabolism and Healthy Volunteers, run by Genzyme, a Sanofi Company at 1 site in United Kingdom.
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 10 inclusion and 25 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 17 Sep 2025. 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 Kingdom. 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?
Percentage of radioactive dose excreted in urine and faeces (time frame: Day -1 up to max Day 43). The study lists 6 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 17 Sep 2025.