Gaucher's Disease
Conditions
Brief summary
The primary objective of the study is to evaluate dose proportionality and pharmacokinetics for three different dose levels of eliglustat after single and repeated administration.
Detailed description
Duration of the study for each subject will be between 42 to 79 days, including a screening period up to 28 days, 3 treatment periods of 7 days each period, a washup period of 7-10 days, and an end-of-study visit 8+/-2 days after the last administration.
Interventions
DRUGEliglustat
Pharmaceutical form:Capsule-Route of administration:Oral
Sponsors
Sanofi
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
* Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m2, inclusive. * Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, electrocardiograms (ECG)). * Having given written informed consent prior to undertaking any study-related procedure * Having given written informed consent prior to undertaking any study-related procedure
Exclusion criteria
Participants are excluded from the study if any of the following criteria apply: * 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 following classes of drugs administered within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy: * Drugs that are strong inducers of CYP3A (eg, rifampin, carbamazepine, phenobarbital,phenytoin, St. John's Wort). * Drugs that inhibit CYP2D6 or CYP3A (eg, paroxetine, ketoconazole, fluconazole,ranitidine). * Drugs that are substrates for P-gp (phenytoin, colchicine and dabigatran etexilate) or CYP2D6 (metoprolol, tricyclic antidepressants such as nortriptyline, amitriptyline, or imipramine, and phenothiazines such as perphenazine and chloropromazine). The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic (PK) parameter: Cmax | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Maximum plasma concentration observed (Cmax) |
| Pharmacokinetic (PK) parameter: tmax | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Time to reach Cmax |
| Pharmacokinetic (PK) parameter: AUC0-T | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve calculated using the trapezoidal method (AUC0-T) |
| Pharmacokinetic (PK) parameter: AUC | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve (AUC) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic (PK) parameter: Ctrough | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Plasma concentration observed just before treatment administration during repeated dosing |
| Pharmacokinetic (PK) parameter: AUClast | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast |
| Number of participants with treatment emergent adverse events, serious adverse events, and adverse event of special interest | Up to Day 42 | Safety was assessed using clinical laboratory evaluations, ECG parameters, and adverse events spontaneously reported by the subject or observed by the Investigator |
| Pharmacokinetic (PK) parameter: tlast | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Time corresponding to the last concentration above the limit of quantification, Clast |
| Pharmacokinetic (PK) parameter: CL/F | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Eliglustat after repeated dose: CL/F |
| Pharmacokinetic (PK) parameter: t1/2z | Multiple timepoints up to Day 35 | Eliglustat after single and repeated doses: Terminal half-life associated with the terminal slope (λz) |
Countries
United States
Outcome results
None listed