A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2

STATUS

Recruiting
participants needed

62
sponsor

Genzyme, a Sanofi Company

Updated on 19 February 2024

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Summary

Primary Objectives:

Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period

Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period

Secondary Objectives:

Primary population:

To assess the effect of venglustat on selected performance test and scale over a 104-week period
To determine the safety and tolerability of venglustat when administered orally once daily over a 104-week period
To assess the pharmacokinetics (PK) of venglustat in plasma and cerebrospinal fluid (CSF)

Secondary population:

To assess the effect of venglustat on selected performance tests and scale over a 104-week period
To determine the safety and tolerability of venglustat when administered once daily over a 104-week period
To assess the PK of venglustat in plasma and CSF

Description

The total duration is up to approximately 119 weeks, including a 60-day screening period, a 104-week treatment period, and a 6-week post-treatment safety observation period.

Details

Condition	Tay-Sachs Disease Sandhoff Disease
Age	2-100 years
Treatment	Placebo, venglustat GZ402671
Clinical Study Identifier	NCT04221451
Sponsor	Genzyme, a Sanofi Company
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Primary population and adult secondary population: age 18 years
	Juvenile/adolescent secondary population: 2 age < 18 years with weight 10 kg
	Participants with a diagnosis of late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) caused by genetic -hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes (primary population only); a secondary population will enroll patients with diagnosis of juvenile/adolescent GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile adult galactosialidosis
	For primary population, the participant has the ability to perform the 9-HPT at the screening visit in < = 240 seconds for the 2 consecutive trials of the dominant hand and the 2 consecutive trials of the nondominant hand
	Participants with a history of seizures well controlled by medication other than strong or moderate inducer or inhibitor of CYP3A4
	Participant is cooperative, able to ingest oral medication, willing to travel to a study site (if applicable), and able to comply with all aspects of the study, including all assessments, according to the Investigator's judgement
	Signed written informed assent/consent
	Contraception for sexually active male participants or female patient; not pregnant or breastfeeding
Exclusion Criteria
	Participant has clinical features of Tay-Sachs or Sandhoff disease, not caused by -hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes and/or is without clinical features
	For primary population and participants with juvenile/adolescent late onset GM2 gangliosidosis and GM1 gangliosidosis, the participant cannot understand and perform all age-appropriate study assessments with the exception of 25FWT
	Relevant medical disorders that would compromise his/her safety
	Documented diagnosis of hepatitis B, C, human immunodeficiency virus 1 or 2
	World Health Organization (WHO) grade >= 2 cortical cataract or a grade >= 2 posterior subcapsular cataract; patients with nuclear cataracts will be accepted
	Participant who requires invasive ventilatory support
	Current treatment by anticoagulants, cataractogenic medications or any medications that may worsen the vision of patient with cataract
	Previous treatment with substrate reduction therapy (SRT) within 3 months prior to study enrollment, strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives prior to enrollment
	Current participation in another study
	Use of investigational medicinal product (IMP) within 3 months or 5 half-lives, whichever is longer, before study enrollment (for N-acetyl-leucine, within 5 half-lives before study enrollment)
	Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin > 2 x the upper limite of normal (ULN) at the time of screening unless the participant has the diagnosis of Gilbert syndrome and maintains a level of bilirubin < 5 mg/dl and direct bilirubin < 20% (1 mg/dl) of total bilirubin level
	Renal insufficiency is defined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 at the screening visit
	The above information is not intended to contain all considerations relevant
	to a patient's potential participation in a clinical trial

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Study Definition

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A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2