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 9 Trials 
21 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
Mozaffar, Tahseen
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
ALKIVIA+, NCT05979441: A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults with Active Idiopathic Inflammatory Myopathy

Enrolling by invitation
3
240
Europe, US, RoW
EFG PH20 SC
argenx
Myositis, Active Idiopathic Inflammatory Myopathy, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
09/27
09/27
ALKIVIA, NCT05523167 / 2021-001277-23: A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Active Idiopathic Inflammatory Myopathy.

Recruiting
2/3
240
Europe, Canada, Japan, US, RoW
EFG PH20 SC, PBO
argenx, argenx BV
Active Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
12/26
02/27
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
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
NCT05669014: A Phase 2 Proof of Concept Study to Evaluate the Efficacy and Safety of Daxdilimab in Participants With Dermatomyositis (DM) or Anti-synthetase Inflammatory Myositis (ASIM)

Active, not recruiting
2
12
Europe, US, RoW
Daxdilimab, HZN-7734, Placebo
Amgen
Idiopathic Inflammatory Myositis
03/25
10/25
RESET-Myositis, NCT06154252: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

Recruiting
1/2
24
Europe, US
CABA-201 following preconditioning with fludarabine and cyclophosphamide
Cabaletta Bio
Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Juvenile Dermatomyositis, Juvenile Polymyositis, Juvenile Idiopathic Inflammatory Myopathy (JIIM), Juvenile Myositis
07/28
07/28
NCT05517603: A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Completed
1/2
25
US
AJ201, JM17, Placebo
AnnJi Pharmaceutical Co., Ltd.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
04/24
04/24
FORTIS, NCT04174105: Gene Transfer Study in Patients With Late Onset Pompe Disease

Recruiting
1/2
18
Europe, US
zocaglusagene nuzaparvovec, AT845
Astellas Gene Therapies
Pompe Disease (Late-onset)
11/29
11/29
LION-CS101, NCT05230459: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting
1/2
10
US
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Asklepios Biopharmaceutical, Inc.
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
12/28
12/28
NCT04093349 / 2019-001283-30: A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Active, not recruiting
1/2
4
Europe, Canada, US
SPK-3006
Spark Therapeutics, Inc.
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
04/32
04/32
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
Avilez, Alyssa
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
Dixon, Stacy
NCT05721573: A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis

Active, not recruiting
2/3
231
Europe, Canada, US, RoW
ABC008
Abcuro, Inc., Syneos Health
Inclusion Body Myositis
11/25
12/25
ALKIVIA, NCT05523167 / 2021-001277-23: A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Active Idiopathic Inflammatory Myopathy.

Recruiting
2/3
240
Europe, Canada, Japan, US, RoW
EFG PH20 SC, PBO
argenx, argenx BV
Active Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
12/26
02/27
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

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