University of Utah - PPDS
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 9 Trials 
16 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Butterfield, Russell
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
HOPE-3, NCT05126758: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, not recruiting
3
104
US
Deramiocel (CAP-1002), Cardiosphere-Derived Cells (CDCs), Placebo
Capricor Inc.
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
12/25
12/27
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
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
NCT04475926: A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

Recruiting
N/A
241
Europe, Canada, US, RoW
Sarepta Therapeutics, Inc.
Limb-girdle Muscular Dystrophy
04/30
04/30
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
Finanger, Erika
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
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
NCT04475926: A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

Recruiting
N/A
241
Europe, Canada, US, RoW
Sarepta Therapeutics, Inc.
Limb-girdle Muscular Dystrophy
04/30
04/30
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
Abdel-Hamid, Hoda Z
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
REACH CDM X, NCT05004129: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Recruiting
2/3
76
Canada, US, RoW
Tideglusib
AMO Pharma Limited
Congenital Myotonic Dystrophy
03/25
03/25
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
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
NCT06550648: Evaluation of the Role of Epstein-Bar Virus in Patients With Pulmonary Arterial Hypertension

Completed
N/A
78
RoW
Right Heart Catheterization (Swan Ganz Catheter)
Cairo University
Pulmonary Arterial Hypertension
03/24
04/24
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

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