LEAP2MONO
Research type
Research Study
Full title
A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT)
IRAS ID
1004975
Contact name
Marine Baixauli
Contact email
Sponsor organisation
Sanofi-aventis recherche & développement
Eudract number
2021-005402-10
Clinicaltrials.gov Identifier
Research summary
The study is called LEAP2MONO and evaluates the safety and effects of an experimental medicine, venglustat, compared to that of another medicine, Cerezyme (imiglucerase), for the treatment of Gaucher Disease Type 3 (GD3).
The purpose of the study is to evaluate a daily oral dose of the study drug known as venglustat and how well it works, especially on the neurological manifestation of GD3, in comparison with Cerezyme. All possible risks related to these two drugs will also be evaluated all along the study. This will be done by one group of participants receiving venglustat & a placebo infusion and comparing them to another group of participants receiving Cereyzme & a placebo tablet for a year. The participants and study doctor will not know which treatment the participant is receiving until the study completes. After the year of blinded treatment, participants will have the opportunity to carry on receiving venglustat until the study completes.
The study will last a minimum of about 2.2 years to a maximum of about 3 years and will include participants aged 12 or older.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
22/ES/0023
Date of REC Opinion
26 Jul 2022
REC opinion
Further Information Unfavourable Opinion