University Health Network_Investigational Site Number : 1240005
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 4 Trials 
10 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Giugliani, Roberto
PERIDOT, NCT05206773 / 2021-002350-90: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Active, not recruiting
3
122
Europe, Canada, Japan, US, RoW
Venglustat (GZ402671), Placebo
Sanofi, Sanofi-Aventis Recherche & Développement
Fabry Disease
12/25
12/26
STARLIGHT, NCT04573023 / 2020-003200-14: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Recruiting
3
80
Europe, US, RoW
JR-141, Idursulfase, JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
01/26
01/26
NCT03566043: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Active, not recruiting
2/3
48
US, RoW
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
11/23
08/25
NCT03632213: Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Active, not recruiting
2
10
RoW
Losartan, Placebo
Hospital de Clinicas de Porto Alegre, The Isaac Foundation
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidoses, MPS IV A, MPS VI, MPS - Mucopolysaccharidosis, Morquio A Syndrome, Morquio Syndrome A, Morquio Syndrome
05/23
08/23
RAINBOW, NCT05758922: Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Active, not recruiting
2
13
RoW
AZ-3102 (Dose 1), Placebo, AZ-3102 (Dose 2)
Azafaros A.G.
GM2 Gangliosidosis, Niemann-Pick Disease, Type C
03/24
12/24
NCT05737784: A Clinical Trial of PRAX-222 in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Recruiting
1/2
60
US, RoW
PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222 - Fixed Doses, Placebo
Praxis Precision Medicines
SCN2A-DEE, Epilepsy
08/25
09/27
PRONTO, NCT05109793: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study

Active, not recruiting
N/A
31
Europe, US, RoW
Azafaros A.G.
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
05/26
05/26
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
N/A
40
Europe, Canada, US, RoW
University of Pennsylvania, Passage Bio, Inc.
GM1 Gangliosidosis
06/26
12/26
Muraro, Juan Marcelo
PERIDOT, NCT05206773 / 2021-002350-90: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Active, not recruiting
3
122
Europe, Canada, Japan, US, RoW
Venglustat (GZ402671), Placebo
Sanofi, Sanofi-Aventis Recherche & Développement
Fabry Disease
12/25
12/26
Phone, Backup
PERIDOT, NCT05206773 / 2021-002350-90: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Active, not recruiting
3
122
Europe, Canada, Japan, US, RoW
Venglustat (GZ402671), Placebo
Sanofi, Sanofi-Aventis Recherche & Développement
Fabry Disease
12/25
12/26

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