Trial record 3 of 11 for:
Skeletal Response to Eliglustat in Patients With Gaucher Disease (EXOSKEL)
This study is enrolling participants by invitation only.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
First received: August 27, 2015
Last updated: July 13, 2017
Last verified: July 2017
Evaluate long term skeletal response to eliglustat in adult patients who successfully completed the Phase 2 or Phase 3 studies.
Evaluate the safety of eliglustat in adult patients who successfully completed the Phase 2 or Phase 3 studies.
||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
||Open Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 Studies
Primary Outcome Measures:
- Change from baseline in bone disease manifestations (clinical, radiological, and biological) [ Time Frame: Baseline to Week 104 ]
Secondary Outcome Measures:
- Assessment of quality of life using Short Form-36 [ Time Frame: Baseline to Week 104 ]
- Measurement of Gaucher disease type I biomarkers [ Time Frame: Baseline to Week 104 ]
- Incidence of adverse events [ Time Frame: Baseline to Week 208 ]
| Estimated Enrollment:
| Actual Study Start Date:
||October 28, 2015
| Estimated Study Completion Date:
||June 22, 2021
| Estimated Primary Completion Date:
||June 22, 2021 (Final data collection date for primary outcome measure)
Cytochrome P450 (CYP) 2D6 Intermediate (IM), Extensive (EM) and Ultra-Rapid (URM) Metaboliser patients will be treated at 84 mg twice daily. CYP2D6 Poor Metabolisers (PM) will be treated at 84 mg once daily.
Route of administration: oral
Other Name: GZ385660
The duration of the study period per patient will be at least 2 years.
|Ages Eligible for Study:
||Child, Adult, Senior
|Sexes Eligible for Study:
|Accepts Healthy Volunteers:
Inclusion criteria :
- The patient must have successfully completed the Phase 2 (GZGD00304) or a Phase 3 study (GZGD02507, GZGD02607 or GZGD03109). Successful completion is defined as patients enrolled in one of the above mentioned studies who received eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely.
- The patient is willing and able to provide signed informed consent prior to any protocol-required procedures being performed.
- Female patients of childbearing potential must have a documented negative pregnancy test prior to enrollment and while they are receiving eliglustat treatment.
- Female patients of childbearing potential must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception (either a barrier method, such as condom or diaphragm + spermicide, or a non-barrier method such as oral, injected, or implanted hormonal methods, or an intra-uterine device or system ) while receiving eliglustat.
- The patient is unwilling to comply with the requirements of the protocol.
- The patient has received an investigational product (other than eliglustat) within 30 days prior to enrollment.
- The patient has received miglustat within the 6 months prior to enrollment.
- The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine transaminase, aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal unless the patient has a diagnosis of Gilbert Syndrome.
- The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation in the study.
- The patient is known to have any of the following: cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or current treatment with Class IA (eg, quinidine) or Class III (eg, amiodarone, sotalol) antiarrhythmic medicinal products.
- The patient has tested positive for the human immunodeficiency virus (HIV) antibody, hepatitis C antibody, or hepatitis B surface antigen.
- The patient has a history of cancer within 6 months of enrolment, with the exception of basal cell carcinoma.
- Patient is CYP 2D6 IM, EM or URM taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor
- Patient is CYP2D6 PM taking a strong CYP3A inhibitor within 2 weeks prior to enrolment.
- If a female patient of childbearing potential has a positive pregnancy test (blood β-Human Chorionic Gonadotropin (β-HCG) or is breast feeding prior to first dosing of eliglustat in this study, the patient cannot enroll in the study at this time, but may be rescreened after the end of the pregnancy, and/or when she is no longer breast feeding, provided rescreening takes place before the end of the enrollment period.
- Women of childbearing potential who are unwilling or unable to be tested for pregnancy.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02536755
|Investigational Site Number 124002
|Montreal, Canada, H3T 1E2 |
|Investigational Site Number 643001
|Moscow, Russian Federation, 125167 |
|Investigational Site Number 643002
|St-Petersburg, Russian Federation, 197341 |
Genzyme, a Sanofi Company
||Clinical Sciences & Operations
||Genzyme, a Sanofi Company
History of Changes
|Other Study ID Numbers:
U1111-1166-6190 ( Other Identifier: UTN )
|Study First Received:
||August 27, 2015
||July 13, 2017
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on August 22, 2017
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Molecular Mechanisms of Pharmacological Action