Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02536911
Other study ID # POP13777
Secondary ID U1111-1170-3678
Status Completed
Phase Phase 1
First received August 28, 2015
Last updated February 9, 2017
Start date September 2015
Est. completion date December 2016

Study information

Verified date February 2017
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Primary Objective:

To study the effect of mild and moderate hepatic 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 and moderate hepatic impairment in comparison with matched subjects with normal hepatic function.


Description:

The total study duration from screening period is approximately 31 days.


Recruitment information / eligibility

Status Completed
Enrollment 24
Est. completion date December 2016
Est. primary completion date December 2016
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years to 79 Years
Eligibility Inclusion criteria :

For subjects with hepatic impairment:

- Male or female subjects, between 18 and 79 years of age, inclusive.

- Body weight between 50.0 and 125.0 kg, inclusive if male, and between 40.0 and 110.0 kg, inclusive if female, body mass index (BMI) between 18.0 and 37 kg/m^2, inclusive.

- Stable chronic liver disease assessed by medical history, physical examination, laboratory values.

- For moderate hepatic impairment cohort: Child-Pugh total score ranging from 7 to 9, inclusive.

- For mild hepatic impairment cohort: Child-Pugh total score ranging from 5 to 6, inclusive.

For matched subjects:

- Male or female subjects, between 18 and 79 years of age, inclusive.

- Body weight within 15% of the body weight of the subjects with hepatic impairment and BMI between 18.0 and 37 kg/m^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).

Exclusion criteria:

For subjects with hepatic impairment:

- Uncontrolled clinically relevant cardiovascular, pulmonary, gastrointestinal, metabolic, hematological, neurological, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.

- Hepatocarcinoma.

- Acute hepatitis.

- Hepatic encephalopathy grade 2, 3, and 4.

- Presence or history of drug hypersensitivity, or allergic disease, including active seasonal rhinitis, diagnosed and treated by a physician.

- History or presence of drug or alcohol abuse (alcohol consumption more than 40 g per day) within 2 years before inclusion.

- If female, pregnancy (defined as positive beta-human chorionic gonadotropin [ß -HCG] blood test), breastfeeding.

- P-gp inhibitors and/or inducers, CYP2D6 and/or CYP3A inhibitors and/or inducers.

- 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 subjects:

- 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 ß-HCG blood test), breastfeeding.

- For subjects 50 years of age and younger: 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 the 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) given within 4 months before inclusion.

- For subjects older than 50 years of age: any significant change in chronic treatment medication within 14 days before inclusion.

- P-gp inhibitors and/or inducers, CYP2D6 and/or CYP3A inhibitors and/or inducers.

- Positive result on any of the following tests: hepatitis B surface antigen (HBs Ag), anti-hepatitis C virus (anti-HCV) antibodies, anti-HIV1 and anti-HIV2 Ab.

- History or presence of drug or alcohol abuse (alcohol consumption more than 40 g per day) within 2 years before inclusion.

- 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
eliglustat
Pharmaceutical form: capsule Route of administration: oral

Locations

Country Name City State
United States Investigational Site Number 840001 Knoxville Tennessee
United States Investigational Site Number 840002 Miami Florida

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Assessment of PK parameter: Maximum plasma concentration observed (Cmax) 3 days
Primary Assessment of PK parameter: Area under the plasma concentration (AUC) 3 days
Secondary Assessment of PK parameter: Area under the plasma concentration versus time curve (AUClast) 3 days
Secondary Assessment of PK parameter: Apparent total body clearance (CL/F) 3 days
Secondary Assessment of PK parameter: Apparent volume of distribution during the terminal phase (Vz/F) 3 days
Secondary Assessment of PK parameter: Predicted accumulation ratio (Rac,pred) 3 days
Secondary Assessment of PK parameter: Terminal half-life (t1/2z) 3 days
Secondary Change from baseline in ECG parameter Baseline, Up to 10 days
Secondary Number of adverse events Up to 10 days
See also
  Status Clinical Trial Phase
Withdrawn NCT04189601 - Complement Activation in the Lysosomal Storage Disorders
Completed NCT04430881 - A National Study in Patients With Unexplained Splenomegaly
Completed NCT02536937 - A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate Phase 1
Completed NCT01411228 - A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Phase 3
Terminated NCT04094181 - A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
Completed NCT00391625 - Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) Phase 1/Phase 2
Completed NCT03625882 - Survey Study for Velaglucerase Alfa (VPRIV) in Japan
Active, not recruiting NCT05526664 - Omics Gaucher Study: Multiomic Approach
Completed NCT02536755 - Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies Phase 3
Recruiting NCT01344096 - Thrombocytopathy in Gaucher Disease Patients N/A
Completed NCT01881633 - A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers Phase 1
Recruiting NCT06116071 - Biomarkers Related to Bone in Pediatric Gaucher Disease
Recruiting NCT01951989 - Intra-monocyte Imiglucerase Kinetics in Gaucher Disease Phase 2
Completed NCT00258778 - Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD) Phase 1
Recruiting NCT04388969 - World Data on Ambroxol for Patients With GD and GBA Related PD
Recruiting NCT05992532 - GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Terminated NCT04145037 - Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease Phase 1/Phase 2
Completed NCT00302146 - Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
Active, not recruiting NCT02605603 - SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
Completed NCT02053896 - A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Phase 2