A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT

Genzyme Corporation0 sites12 target enrollmentMarch 11, 2021

Overview

No summary available.

Registry

who.int

Start Date

March 11, 2021

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•\- GD3 adult participant \=18 years of age.
•\- GD3 pediatric participant is \=12 years of age \<18 years and \=30 kg of weight. For male patients 12 to \<18 years of age: There is a potential risk for venglustat to impact male fertility particularly when used in patients who have not reached a certain stage of sexual maturation. If a patient has not reached Tanner Stage 3 by the screening visit, the Investigator should assess and discuss with the patient the potential benefits and risks when considering eligibility for study participation. In this benefit/risk assessment\-discussion, the parent(s)/legal guardian(s), the patient (if considered able by local regulation), and the Sponsor’s medical representative (if considered appropriate) shall be consulted.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1\.2 Adults (18\-64 years) yes
•F.1\.2\.1 Number of subjects for this age range 24
•F.1\.3 Elderly (\>\=65 years) no
•F.1\.3\.1 Number of subjects for this age range

•\- Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.
•\- Participant has had a partial or total splenectomy within 3 years prior to randomization.
•\- Participant is blood transfusion\-dependent.
•\- Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase \[ALT]/ aspartate aminotransferase \[AST]) or total bilirubin \>2 times the upper limit of normal, unless
•the patient has a diagnosis of Gilbert Syndrome.
•\- Participant has any clinically significant disease, other than GD, including cardiovascular (congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect), hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia) or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation.
•\- Participant has renal insufficiency, as defined by an estimated glomerular filtration rate \<30 mL/min/1\.73m2 at the screening visit.
•\- Participant has received an investigational product within 30 days prior to enrollment.
•\- Participant has a history of cancer, with the exception of basal cell carcinoma.
•\- Participant has myoclonic seizures.