University of California Irvine, Department of Neurology
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 6 Trials 
14 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
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
Scott, Jennifer
NCT05211986: Safety and Tolerability of IMM01-STEM in Patients With Muscle Atrophy Related to Knee Osteoarthritis.

Completed
1/2
13
US
IMM01-STEM
Immunis, Inc.
Muscle Atrophy
08/24
11/24
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

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