University of Connecticut/Connecticut Children's Medical Center
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 13 Trials 
27 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Day, John W
RESPOND, NCT04488133 / 2020-003492-18: A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Active, not recruiting
4
46
Europe, US, RoW
Nusinersen, ISIS 396443, BIIB058, Spinraza
Biogen
Muscular Atrophy, Spinal
10/25
10/25
DEVOTE, NCT04089566 / 2019-002663-10: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Completed
3
145
Europe, Canada, Japan, US, RoW
Nusinersen, BIIB058
Biogen
Muscular Atrophy, Spinal
02/24
05/24
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
REACH CDM, NCT03692312: Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Completed
2/3
56
Europe, Canada, US, RoW
Tideglusib, Placebo
AMO Pharma Limited
Congenital Myotonic Dystrophy
04/23
04/23
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
ALSpire, NCT04494256 / 2020-000207-36: A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation

Terminated
1/2
99
Europe, Canada, US
BIIB105, Placebo
Biogen, Biogen Idec Research Limited
Amyotrophic Lateral Sclerosis
08/24
08/24
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
NCT06089018: Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

Active, not recruiting
N/A
125
US
Stanford University, Biogen
Myotonic Dystrophy 1
09/24
09/24
INC-6603, NCT01203085: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Recruiting
N/A
500
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, Children's Hospital of Philadelphia, University College London Hospitals, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Sydney Children's Hospitals Network, Nemours Children's Clinic
Charcot Marie Tooth Disease
12/24
12/24
INC-6601, NCT01193075: Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Recruiting
N/A
5000
Europe, Canada, US, RoW
Michael Shy, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Johns Hopkins University, National Institute of Neurological Disorders and Stroke (NINDS), King's College Hospital NHS Trust, Nemours Children's Hospital, Stanford University, University of Pennsylvania, University of Rochester, Children's Hospital of Philadelphia, Sydney Children's Hospitals Network, Rare Diseases Clinical Research Network, Muscular Dystrophy Association, National Institutes of Health (NIH), Charcot-Marie-Tooth Association, Massachusetts General Hospital, Cedars-Sinai Medical Center, University of Miami, University of Minnesota, Connecticut Children's Medical Center, University of Colorado, Denver, The National Hospital for Neurology and Neurosurgery, Dubowitz Neuromuscular Centre
Charcot Marie Tooth Disease
12/26
12/26
INC-6602, NCT01193088: Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Recruiting
N/A
1050
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, University of Pennsylvania, King's College Hospital NHS Trust, Sydney Children's Hospitals Network, Children's Hospital of Philadelphia, University of Miami, Johns Hopkins University, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Cedars-Sinai Medical Center, Nemours Children's Clinic, Stanford University, University of Minnesota, Massachusetts General Hospital, University of Colorado, Denver, Children's National Research Institute, University of Michigan, St. Jude Children's Research Hospital, Connecticut Children's Medical Center, Seattle Children's Hospital, The Hospital for Sick Children
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
12/26
12/26
NCT03981575: Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Recruiting
N/A
700
Europe, Canada, US, RoW
Virginia Commonwealth University, University of Rochester, Stanford University, Ohio State University, University of Florida, University of Iowa, Ludwig-Maximilians - University of Munich, Fondazione Serena Onlus - Centro Clinico NeMO Milano, The Methodist Hospital Research Institute, Radboud University Medical Center, University College London Hospitals, University of California, Los Angeles
Myotonic Dystrophy 1, DM1
10/26
12/26
NCT06774703: Nemaline Myopathy Clinical Research Network (NM-CTRN)

Not yet recruiting
N/A
50
Canada, US
Stanford University, A Foundation Building Strength
Nemaline Myopathy
12/29
12/29
ASPIRE-DM1, NCT05224778: DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Recruiting
N/A
50
Europe, US
Virginia Commonwealth University
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, CDM, CHDM
10/26
12/26
NCT05434650: Abbott Atrial Fibrillation Post Approval Study

Active, not recruiting
N/A
200
US
electrophysiology study and ablation with an Abbott Ablation Catheter
Abbott Medical Devices
Atrial Arrhythmia, Atrium; Fibrillation, Atrial Tachycardia
10/24
11/26
Acsadi, Gyula
STEER, NCT05089656: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Active, not recruiting
3
127
Europe, US, RoW
OAV101, Zolgensma, AVXS-101, Sham control
Novartis Pharmaceuticals
Type 2 Spinal Muscular Atrophy
11/24
02/25
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
INC-6603, NCT01203085: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Recruiting
N/A
500
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, Children's Hospital of Philadelphia, University College London Hospitals, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Sydney Children's Hospitals Network, Nemours Children's Clinic
Charcot Marie Tooth Disease
12/24
12/24
INC-6601, NCT01193075: Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Recruiting
N/A
5000
Europe, Canada, US, RoW
Michael Shy, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Johns Hopkins University, National Institute of Neurological Disorders and Stroke (NINDS), King's College Hospital NHS Trust, Nemours Children's Hospital, Stanford University, University of Pennsylvania, University of Rochester, Children's Hospital of Philadelphia, Sydney Children's Hospitals Network, Rare Diseases Clinical Research Network, Muscular Dystrophy Association, National Institutes of Health (NIH), Charcot-Marie-Tooth Association, Massachusetts General Hospital, Cedars-Sinai Medical Center, University of Miami, University of Minnesota, Connecticut Children's Medical Center, University of Colorado, Denver, The National Hospital for Neurology and Neurosurgery, Dubowitz Neuromuscular Centre
Charcot Marie Tooth Disease
12/26
12/26
INC-6602, NCT01193088: Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Recruiting
N/A
1050
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, University of Pennsylvania, King's College Hospital NHS Trust, Sydney Children's Hospitals Network, Children's Hospital of Philadelphia, University of Miami, Johns Hopkins University, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Cedars-Sinai Medical Center, Nemours Children's Clinic, Stanford University, University of Minnesota, Massachusetts General Hospital, University of Colorado, Denver, Children's National Research Institute, University of Michigan, St. Jude Children's Research Hospital, Connecticut Children's Medical Center, Seattle Children's Hospital, The Hospital for Sick Children
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
12/26
12/26
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting
N/A
700
Europe, Japan, US, RoW
Prospective observational registry, Zolgensma
Novartis Pharmaceuticals, United BioSource, LLC
Spinal Muscular Atrophy (SMA)
06/38
06/38
Kosikowski, Ashley
INC-6603, NCT01203085: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Recruiting
N/A
500
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, Children's Hospital of Philadelphia, University College London Hospitals, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Sydney Children's Hospitals Network, Nemours Children's Clinic
Charcot Marie Tooth Disease
12/24
12/24
INC-6601, NCT01193075: Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Recruiting
N/A
5000
Europe, Canada, US, RoW
Michael Shy, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Johns Hopkins University, National Institute of Neurological Disorders and Stroke (NINDS), King's College Hospital NHS Trust, Nemours Children's Hospital, Stanford University, University of Pennsylvania, University of Rochester, Children's Hospital of Philadelphia, Sydney Children's Hospitals Network, Rare Diseases Clinical Research Network, Muscular Dystrophy Association, National Institutes of Health (NIH), Charcot-Marie-Tooth Association, Massachusetts General Hospital, Cedars-Sinai Medical Center, University of Miami, University of Minnesota, Connecticut Children's Medical Center, University of Colorado, Denver, The National Hospital for Neurology and Neurosurgery, Dubowitz Neuromuscular Centre
Charcot Marie Tooth Disease
12/26
12/26
INC-6602, NCT01193088: Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Recruiting
N/A
1050
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, University of Pennsylvania, King's College Hospital NHS Trust, Sydney Children's Hospitals Network, Children's Hospital of Philadelphia, University of Miami, Johns Hopkins University, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Cedars-Sinai Medical Center, Nemours Children's Clinic, Stanford University, University of Minnesota, Massachusetts General Hospital, University of Colorado, Denver, Children's National Research Institute, University of Michigan, St. Jude Children's Research Hospital, Connecticut Children's Medical Center, Seattle Children's Hospital, The Hospital for Sick Children
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
12/26
12/26
Twatchtman-Bassett, Jennifer
INC-6603, NCT01203085: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Recruiting
N/A
500
Europe, US, RoW
University of Iowa, National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association, University of Rochester, Children's Hospital of Philadelphia, University College London Hospitals, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Sydney Children's Hospitals Network, Nemours Children's Clinic
Charcot Marie Tooth Disease
12/24
12/24
INC-6601, NCT01193075: Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Recruiting
N/A
5000
Europe, Canada, US, RoW
Michael Shy, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Johns Hopkins University, National Institute of Neurological Disorders and Stroke (NINDS), King's College Hospital NHS Trust, Nemours Children's Hospital, Stanford University, University of Pennsylvania, University of Rochester, Children's Hospital of Philadelphia, Sydney Children's Hospitals Network, Rare Diseases Clinical Research Network, Muscular Dystrophy Association, National Institutes of Health (NIH), Charcot-Marie-Tooth Association, Massachusetts General Hospital, Cedars-Sinai Medical Center, University of Miami, University of Minnesota, Connecticut Children's Medical Center, University of Colorado, Denver, The National Hospital for Neurology and Neurosurgery, Dubowitz Neuromuscular Centre
Charcot Marie Tooth Disease
12/26
12/26

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