This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and \<18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.
Screening period: 45 days Double blind, double-dummy, primary analysis treatment period: 52 weeks Open label extended treatment period: minimum of 52 weeks due to a common study end of treatment date Follow up phone call: 30-37 days after end of treatment
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
43
tablet; oral
sterile lyophilized product; intravenous
Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score
Time frame: From baseline to Week 52
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score
Time frame: From baseline to Week 52
Percent change in spleen volume
Time frame: From baseline to Week 52
Percent change in liver volume
Time frame: From baseline to Week 52
Change in hemoglobin level
Time frame: From baseline to Week 52
Percent change in platelet count
Time frame: From baseline to Week 52
Percent change in CSF GL-1 and lyso-GL-1 levels
Time frame: From baseline to Week 52
Percent change in plasma GL-1 and lyso-GL-1 levels
Time frame: From baseline to Week 52
Number of patients with treatment emergent adverse events (TEAEs)/ serious adverse events (SAEs)/ adverse events of special interest (AESIs)
Time frame: From baseline to max of 4.5 years
Change in score of Beck Depression Inventory II (BDI-II) during the treatment-emergent period (for participants 13 years of age and above at baseline)
Time frame: From baseline to Week 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Yale University School of Medicine - Investigational Site Number: 8400003
New Haven, Connecticut, United States
University of Iowa - Investigational Site Number: 8400002
Iowa City, Iowa, United States
Texas Oncology - Medical City Dallas Site Number : 8400008
Dallas, Texas, United States
Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001
Fairfax, Virginia, United States
Hospital de Ninos - Investigational Site Number: 320001
Buenos Aires, Argentina
Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001
Winnipeg, Manitoba, Canada
National Taiwan University Hospital-Investigational Site Number: 1580001
Taipei, Taiwan, China
Peking Union Medical College Hospital - Investigational Site Number: 1560001
Beijing, China
The First Affiliated Hospital - Investigational Site Number: 1560002
Guangzhou, China
Xinhua Hospital - Investigational Site Number: 1560004
Shanghai, China
...and 12 more locations
Change in Patient Health Questionnaire 9 (PHQ-9) during the treatment-emergent period (for participants 12 years of age at baseline)
Time frame: From baseline to Week 52