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 14 Trials 
10 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Wicklund, Matthew
Fortify, NCT05775848: Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

Active, not recruiting
3
81
Europe, US, RoW
BBP-418 (ribitol), Placebo
ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
07/27
07/27
NCT04886518: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Hourglass Jul 2022 - Dec 2022 : Efficacy and safety data for treatment of EDS in patients with Myotonic Dystrophy Type 1
Active, not recruiting
2
30
Canada, US
Pitolisant Oral Tablet, Placebo oral tablet
Harmony Biosciences, LLC
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
10/23
10/24
MOVE FSHD, NCT04635891: Motor Outcomes to Validate Evaluations in FSHD

Recruiting
N/A
450
Europe, Canada, US
University of Kansas Medical Center, FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche
FSHD
12/26
01/27
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
Menard-Katcher, Paul
DEGS, NCT03678545: Dupilumab in Eosinophilic Gastritis

Active, not recruiting
2
41
US
Dupilumab (blinded), Placebo (blinded), Dupilumab (open-label)
Children's Hospital Medical Center, Cincinnati, Regeneron Pharmaceuticals, National Institutes of Health (NIH)
Eosinophilic Gastritis, Eosinophilic Gastroenteritis
03/24
11/24
Blume, Brianna
REAL, NCT05218668: Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis

Active, not recruiting
2
40
US, RoW
Fasudil (WP-0512)
Woolsey Pharmaceuticals
Amyotrophic Lateral Sclerosis
07/26
01/28
NCT04146051: Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)

Recruiting
2
30
Canada, US
Descartes-08
Cartesian Therapeutics
Myasthenia Gravis, Generalized
03/25
03/26
NCT06069934: Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

No Longer Available
N/A
US
Pridopidine
Prilenia, Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
Amyotrophic Lateral Sclerosis
 
 
Cahoon, Jonathon
DEGS, NCT03678545: Dupilumab in Eosinophilic Gastritis

Active, not recruiting
2
41
US
Dupilumab (blinded), Placebo (blinded), Dupilumab (open-label)
Children's Hospital Medical Center, Cincinnati, Regeneron Pharmaceuticals, National Institutes of Health (NIH)
Eosinophilic Gastritis, Eosinophilic Gastroenteritis
03/24
11/24
Baines, Brenna
NCT04886518: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Hourglass Jul 2022 - Dec 2022 : Efficacy and safety data for treatment of EDS in patients with Myotonic Dystrophy Type 1
Active, not recruiting
2
30
Canada, US
Pitolisant Oral Tablet, Placebo oral tablet
Harmony Biosciences, LLC
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
10/23
10/24

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