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

   

 
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
GRASP, NCT03981289: Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

Active, not recruiting
N/A
80
Europe, US
Virginia Commonwealth University, Newcastle University, Nationwide Children's Hospital, Washington University School of Medicine, University of Iowa, University of Minnesota, University of California, Irvine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc., University of Colorado, Denver, University of Kansas Medical Center
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
06/25
06/25
INSPIRE-IBM, NCT05046821: Sporadic Inclusion Body Myositis Natural History Study

Active, not recruiting
N/A
150
US
University of California, Irvine, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Sporadic Inclusion Body Myositis, Inclusion Body Myositis
01/26
01/26
GRASP-01-003, NCT05618080: LGMD R1 Natural History Study

Recruiting
N/A
100
Europe, US
Virginia Commonwealth University, Nationwide Children's Hospital, Washington University School of Medicine, University of Iowa, University of Florida, University of Minnesota, Newcastle University, University of Kansas Medical Center, University of Colorado, Denver, Indiana CHC (Community Health Clinic), University of California, Irvine
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
05/28
08/28
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
COPE, NCT06093737: Strengthening Community Mental Health

Recruiting
N/A
300
US
COPE: Communities Organizing for Power through Empathy
Louisiana State University and A&M College, National Academies of Sciences, Engineering and Medicine, University of Illinois at Urbana-Champaign
Mental Health, Emotional Wellbeing, Anxiety, Depression, Stress, Coping, Perceived Stress, Perceived Social Support
06/25
06/25

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