Medical Genetics Service/HCPA, Department of Genetics/UFRGS
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 5 Trials 
8 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

Recruiting
3
114
Europe, Canada, Japan, US, RoW
Venglustat (GZ402671), Placebo
Sanofi, Sanofi-Aventis Recherche & Développement
Fabry Disease
11/24
10/25
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)
05/24
05/25
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
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
Silva, Larissa Da
NCT06214637: Effectiveness of Melissa Officinalis for the Treatment of Probable Sleep Bruxism in Children and Adolescents

Recruiting
1
42
RoW
Melissa, Placebo
Universidade Federal do Rio de Janeiro
Sleep Bruxism, Adolescent, Children, Temporomandibular Joint Disorders
08/24
09/24

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