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Gaucher's Disease Type IPhase 1NCT06143904

A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

A Phase 1 study testing Genz-112638 and [14C]-Genz-112638 in people with Gaucher's Disease Type I, run by Sanofi.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Objectives: To determine pharmacokinetic (PK) variables, including absolute bioavailability (F), of Genz-99067, the free base of the L-tartaric acid salt of Genz-112638 as it exists in plasma, after a single intravenous (IV) dose and after a single oral dose of Genz-112638 (unlabeled). Read more about this study

Who is it for?
Ages 18 to 45 years, men onlyHealthy volunteers can take part
Conditions the study is for
Gaucher's Disease Type I
What is being tested?
Genz-112638 and [14C]-Genz-112638
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
No sites listed yet
Study dates
3 Jun 2009to 5 Jul 2009
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 45 years
  • Men only
  • Healthy volunteers accepted
  • Adult

Inclusion Criteria: Having given written informed consent prior to undertaking any study-related procedure The subject has a body weight of 50 to 100 kg [110 to 220 pounds (lb)] with a body mass index (BMI) less than 30 kilograms per square meter (kg/m2) at Screening.

The subject's physical examination results, vital signs, laboratory assessments, and cardiac assessments are within normal limits or, if abnormal, are not clinically significant at Screening and Day -1. Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:

Prolonged QTc interval (eg, repeated demonstration of a QTc interval ≥450 msec), family history of long QT or Brugada Syndrome, and/or history of sudden death in a first-degree relative.

The subject receives an immunization within 30 days of providing informed consent.

The subject has a history of drug allergies (eg, significant rash, hives, etc in response to antibiotics).

The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.

About this study

Official title: An Open-Label Study to Evaluate the Absolute Bioavailability of Genz-112638 and the Absorption, Metabolism, and Excretion of [14C]-Genz-112638 in Male Subjects

Objectives:

To determine pharmacokinetic (PK) variables, including absolute bioavailability (F), of Genz-99067, the free base of the L-tartaric acid salt of Genz-112638 as it exists in plasma, after a single intravenous (IV) dose and after a single oral dose of Genz-112638 (unlabeled).

To determine the PK, total recovery, routes and rates of excretion, and the metabolic profile of Genz-99067 after 5 days of BID oral dosing with unlabeled Genz-112638 followed by a single dose of [14C]-Genz-112638.

Read the detailed description

The maximum study duration for an individual subject was approximately 65 days from the beginning of Screening through the Safety Follow-up visit.

Being tested
Genz-112638, [14C]-Genz-112638
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Genz-112638Drug

    Also called GZ385660/ eliglustat, Cerdelga

    Pharmaceutical form:Solution-Route of administration:IV

  • Genz-112638Drug

    Also called GZ385660/ eliglustat

    Pharmaceutical form:Capsule-Route of administration:Oral

  • [14C]-Genz-112638Drug

    Also called GZ385660/ eliglustat

    Pharmaceutical form:Solution-Route of administration:Oral

Study groups in NCT06143904
Study groupRoleWhat they receive
Treatment Period 1 -2ExperimentalSingle-dose Intravenous (IV) Genz-112638 Day 1 followed by single dose GenZ-112638 oral capsules Day 8
Receives: Drug: Genz-112638
Treatment Period 1-4ExperimentalSingle-dose Intravenous (IV) Genz-112638 Day 1; single dose GenZ-112638 oral capsules Day 8; oral capsule Genz-112638 Days 9-14; single dose [14C]-Genz-112638 oral solution Day 15
Receives: Drug: Genz-112638; Drug: [14C]-Genz-112638

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Assessment of pharmacokinetic (PK) parameter: Cmax

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  2. Assessment of pharmacokinetic (PK) parameter: Tmax

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  3. Assessment of pharmacokinetic (PK) parameter: AUC0-∞

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  4. Assessment of pharmacokinetic (PK) parameter: AUC0 -τ

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  5. Assessment of pharmacokinetic (PK) parameter: AUC0-∞/D

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  6. Assessment of pharmacokinetic (PK) parameter: AUC0-τ/D

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  7. Assessment of pharmacokinetic (PK) parameter: F

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  8. Assessment of pharmacokinetic (PK) parameter: CL/F

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  9. Assessment of pharmacokinetic (PK) parameter: t½

    Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

    Measured: Multiple timepoints up to Day 26

  10. Pharmacokinetic (PK) parameter: Absolute bioavailability (F) of single-dose oral versus single-dose IV administration

    Measured: Multiple timepoints up to Day 26

  11. Assessment of pharmacokinetic (PK) parameter: Total radioactivity excreted in urine and feces

    Total radioactivity excreted in urine and feces will be converted to percentage of radioactive dose.

    Measured: Multiple timepoints up to Day 26

  12. Assessment of pharmacokinetic (PK) parameter: Total radioactivity in whole blood and plasma

    Total radioactivity in whole blood and plasma will be converted to ngEq/g Genz-99067 concentration for whole blood and ngEq/mL for plasma, based on the dose specific activity.

    Measured: Multiple timepoints up to Day 26

  13. Assessment of pharmacokinetic (PK) parameter: % relative abundance of each component in samples of plasma or excreta

    It will be estimated as [(radioactivity for the HPLC peak)/(total radioactivity injected per HPLC run) x 100].

    Measured: Multiple timepoints up to Day 26

  14. Assessment of pharmacokinetic (PK) parameter: The percentage of the administered dose attributed to each component in samples of urine or feces

    It will be estimated as [(% relative abundance)/100 x (percentage of radioactive dose in the sample)].

    Measured: Multiple timepoints up to Day 26

  15. Assessment of pharmacokinetic (PK) parameter: The radioactivity of [14C]-Genz-99067 and each major metabolite in plasma, as identified by radio-profiling

    It will be converted to equivalent concentrations as [(% relative abundance)/100 x (equivalent concentration of total radioactivity in the sample)].

    Measured: Multiple timepoints up to Day 26

  16. Noncompartmental PK parameters: AUC0-t

    Measured: Multiple timepoints up to Day 26

  17. Noncompartmental PK parameters: AUC0-∞

    Measured: Multiple timepoints up to Day 26

  18. Noncompartmental PK parameters: Cmax

    Measured: Multiple timepoints up to Day 26

  19. Noncompartmental PK parameters: Tmax

    Measured: Multiple timepoints up to Day 26

  20. Noncompartmental PK parameters: t½

    Measured: Multiple timepoints up to Day 26

  21. Noncompartmental PK parameters: Vz/F

    Measured: Multiple timepoints up to Day 26

  22. Noncompartmental PK parameters: CL/F

    Measured: Multiple timepoints up to Day 26

  23. Noncompartmental PK parameters for urine and feces: Cum Ae

    Measured: Multiple timepoints up to Day 26

  24. Noncompartmental PK parameters for urine and feces: % dose

    Measured: Multiple timepoints up to Day 26

  25. Renal clearance (CLR) for total plasma radioactivity and Genz-99067

    Measured: Multiple timepoints up to Day 26

  26. PK parameters [AUC0-τ, AUC0-∞, Cmax, Tmax, t½] and metabolite ratio for metabolite(s) of Genz-99067

    Measured: Multiple timepoints up to Day 26

Secondary outcomes (1)

  1. Number of participants with treatment-emergent adverse events (TEAEs), serious adverse event (SAEs), and adverse event of special interest (AESI)

    Measured: Up to Day 33

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Nov 2023
  2. Study start3 Jun 2009
  3. Primary completion5 Jul 2009
  4. Study completion5 Jul 2009

Resources

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
Basic science
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06143904
Sponsor's protocol ID
GZGD02107
Other IDs
U1111-1294-7994
Status last verified
Nov 2023
First submitted
16 Nov 2023
Last updated
22 Nov 2023
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

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Genz-112638 and [14C]-Genz-112638 in people with Gaucher's Disease Type I, run by Sanofi.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 22 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.

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) parameter: Cmax (time frame: Multiple timepoints up to Day 26). The study lists 26 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06143904.

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 22 Nov 2023.

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