A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00358150
• Date of registration: 27/07/2006
• Prospective Registration: No
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
• Scientific title: A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients
• Date of first enrolment: June 2006
• Target sample size: 26
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00358150
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Argentina, Israel, Italy, Mexico, Russian Federation, United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

Inclusion Criteria:

- The participant had a diagnosis of Gaucher Type I disease and a documented deficiency
of glucocerebrosidase activity by enzyme assay and was willing and able to provide
written informed consent prior to initiating any study-related procedures;

- The participant was 18 to 65 years old and weighed between 50 and 120 kilogram (kg) at
enrollment;

- The participant had the following symptoms of Gaucher disease identified within 28
days of enrollment (at screening);

- Anemia - indicated by hemoglobin measurements taken during the screening phase (8
to 10 gram per deciliter (g/dL) if female, 8 to 11 g/dL if male);

- Thrombocytopenia - indicated by platelet count measurements taken during the
screening phase (60000 to 100000 per cubic millimeter);

- Splenomegaly, as indicated by magnetic resonance imaging (MRI) or spiral computed
tomography (CT) (>= 10 multiples of normal);

- Female participants of child-bearing potential must had a documented negative serum
pregnancy test prior to dosing. Female participants agreed to use a reliable method of
birth control throughout duration of trial.

Exclusion Criteria:

- Participant had a partial or total splenectomy or infarcted areas of the spleen;

- Participant had documented prior bleeding varices or liver infarction;

- Participant received miglustat within 12 months prior to study enrollment;

- The participant had received an investigational product within 30 days prior to study
enrollment;

- Participant had neurologic or pulmonary involvement;

- Participant had new pathological bone involvement or bone crisis in the 12 months
prior to enrollment;

- Participant was transfusion-dependent;

- Participant had a documented etiology of anemia due to causes other than Gaucher
disease;

- The participant had cardiac functional and/or anatomical abnormalities, a history of
cancer or tested positive for human immunodeficiency virus (HIV) antibody or
Hepatitis;

- Participant had a clinically significant disease, other than Gaucher disease,
including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic,
endocrine, metabolic, or psychiatric disease, other medical conditions, or serious
intercurrent illnesses that, in the opinion of the Investigator, might preclude
participation in the study.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cerebroside Lipidosis Syndrome

Gaucher Disease, Non-Neuronopathic Form

Glucocerebrosidase Deficiency Disease

Glucosylceramide Beta-Glucosidase Deficiency Disease

Gaucher Disease, Type 1

Intervention(s)

Drug: Eliglustat tartrate

Primary Outcome(s)

Percentage of Participants Demonstrating A Meaningful Clinical Response [Time Frame: Baseline, Year 1]

Secondary Outcome(s)

Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS) [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)]

Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2 [Time Frame: Baseline, Year 1, Year 2]

Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)]

Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)]

Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)]

Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)]

Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study [Time Frame: Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)]

Secondary ID(s)

2005-004732-42

GZGD00304

DRI12816

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/09/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00358150