University of Iowa Children's Hospital
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 24 Trials 
38 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Colaizy, Tarah T
MIDAS, NCT06679855: Milrinone for Prevention of Post-ligation Cardiac Syndrome Trial

Not yet recruiting
3
316
US
Milrinone infusion, Placebo infusion
NICHD Neonatal Research Network, National Heart, Lung, and Blood Institute (NHLBI)
Post-ligation Cardiac Syndrome
06/27
08/29
NCT00063063: Generic Database of Very Low Birth Weight Infants

Recruiting
N/A
80000
US
NICHD Neonatal Research Network, National Center for Research Resources (NCRR), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
03/30
03/30
Mathews, Katherine
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
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
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
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
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
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
NCT00313677: Clinical Trial Readiness for the Dystroglycanopathies

Recruiting
N/A
190
US
Katherine Mathews, National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy
07/25
07/26
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
FA-COMS, NCT03090789: FA Clinical Outcome Measures

Active, not recruiting
N/A
2000
Canada, US, RoW
Friedreich's Ataxia Research Alliance, University of Rochester, Children's Hospital of Philadelphia
Friedreich Ataxia, Neuro-Degenerative Disease
01/30
01/30
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
UNIFAI, NCT06016946: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Recruiting
N/A
3000
Europe, Canada, US, RoW
Friedreich's Ataxia Research Alliance
Friedreich Ataxia
01/48
01/48
Flaherman, Valerie
PRIMES, NCT04704076: Preventing Infant Malnutrition With Early Supplementation

Completed
N/A
324
RoW
ESVS, Exclusive breastfeeding
University of California, San Francisco, International Partnership for Human Development, Makerere University, Bill and Melinda Gates Foundation, University of California, Davis, University of Aberdeen
Underweight, Wasting, Stunting, Breastfeeding, Microbial Colonization
01/22
01/23
NCT05172011: Understanding the Long-term Impact of COVID on Children and Families

Active, not recruiting
N/A
15028
US
NYU Langone Health, National Heart, Lung, and Blood Institute (NHLBI)
SARS-CoV-2 Infection
04/27
05/27
Griffin, Jennifer
NuPower, NCT05162768 / 2021-003907-16: Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

Active, not recruiting
3
102
Europe, US, RoW
Elamipretide, MTP-131, Placebo
Stealth BioTherapeutics Inc.
Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
09/24
10/24
Kair, Laura R
NCT03901833: LIFT: Telemedicine Breastfeeding Support

Completed
N/A
56
US
Telemedicine Support, Standard of Care
University of California, Davis, National Institutes of Health (NIH)
Breastfeeding
03/23
03/24
NCT05282394: Newborn Abdominal Massage to Prevent Hyperbilirubinemia

Recruiting
N/A
30
US
Abdominal massage, Reading
University of California, Davis, National Institutes of Health (NIH), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Newborn Jaundice
12/24
12/24
Reinking, Benjamin
NPC-QIC, NCT02852031: National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Recruiting
N/A
1000
Europe, Canada, US
Collaborative Learning Network
Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome (HLHS)
05/26
05/28
Stephan, Carrie
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
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
NCT00313677: Clinical Trial Readiness for the Dystroglycanopathies

Recruiting
N/A
190
US
Katherine Mathews, National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy
07/25
07/26
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
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
Maldonado, Jennifer
AT-PATCH, NCT03209583: ATrial Tachycardia PAcing Therapy in Congenital Heart

Recruiting
N/A
300
Canada, US
Medtronic
Ian Law, Memorial Healthcare System, Children's Hospital of Michigan, Indiana University Health, Mayo Clinic, Rainbow Babies and Children's Hospital, The Hospital for Sick Children, University of Wisconsin, Madison, University of California, Los Angeles, University of Utah, Norton Healthcare, University of Michigan, Vanderbilt University Medical Center, Children's Hospital of Orange County
Atrial Arrhythmia, Atrial Tachycardia, Congenital Heart Disease, Pacemaker Re-Entrant Tachycardia
09/26
09/27
Pierick, Trudy
NPC-QIC, NCT02852031: National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Recruiting
N/A
1000
Europe, Canada, US
Collaborative Learning Network
Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome (HLHS)
05/26
05/28
McDaniel, Corey
FA-COMS, NCT03090789: FA Clinical Outcome Measures

Active, not recruiting
N/A
2000
Canada, US, RoW
Friedreich's Ataxia Research Alliance, University of Rochester, Children's Hospital of Philadelphia
Friedreich Ataxia, Neuro-Degenerative Disease
01/30
01/30
UNIFAI, NCT06016946: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Recruiting
N/A
3000
Europe, Canada, US, RoW
Friedreich's Ataxia Research Alliance
Friedreich Ataxia
01/48
01/48
Pham, Tina
AT-PATCH, NCT03209583: ATrial Tachycardia PAcing Therapy in Congenital Heart

Recruiting
N/A
300
Canada, US
Medtronic
Ian Law, Memorial Healthcare System, Children's Hospital of Michigan, Indiana University Health, Mayo Clinic, Rainbow Babies and Children's Hospital, The Hospital for Sick Children, University of Wisconsin, Madison, University of California, Los Angeles, University of Utah, Norton Healthcare, University of Michigan, Vanderbilt University Medical Center, Children's Hospital of Orange County
Atrial Arrhythmia, Atrial Tachycardia, Congenital Heart Disease, Pacemaker Re-Entrant Tachycardia
09/26
09/27
Nayar, Shakti
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
NCT03709784: Spinraza in Adult Spinal Muscular Atrophy

Recruiting
N/A
148
Canada, US
Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
Washington University School of Medicine
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
12/24
01/25
REFINE-ALS, NCT04259255: Radicava® (Edaravone) Findings in Biomarkers From ALS

Active, not recruiting
N/A
300
Canada, US
Edaravone (Radicava®/Radicava ORS®), Radicava®, Radicava ORS®
Mitsubishi Tanabe Pharma America Inc., Massachusetts General Hospital
Amyotrophic Lateral Sclerosis, ALS
03/24
03/24
Grippo, Erica Del
NPC-QIC, NCT02852031: National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Recruiting
N/A
1000
Europe, Canada, US
Collaborative Learning Network
Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome (HLHS)
05/26
05/28
Riley, Emily
AT-PATCH, NCT03209583: ATrial Tachycardia PAcing Therapy in Congenital Heart

Recruiting
N/A
300
Canada, US
Medtronic
Ian Law, Memorial Healthcare System, Children's Hospital of Michigan, Indiana University Health, Mayo Clinic, Rainbow Babies and Children's Hospital, The Hospital for Sick Children, University of Wisconsin, Madison, University of California, Los Angeles, University of Utah, Norton Healthcare, University of Michigan, Vanderbilt University Medical Center, Children's Hospital of Orange County
Atrial Arrhythmia, Atrial Tachycardia, Congenital Heart Disease, Pacemaker Re-Entrant Tachycardia
09/26
09/27

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