UCL Great Ormond Street Institute of Child Health
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 19 Trials 
34 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Jackson, Christopher G
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05549258 / 2021-003528-33: Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

Recruiting
2
15
Europe, Canada, US, RoW
Inebilizumab
Amgen, Horizon Therapeutics Ireland DAC
Neuromyelitis Optica Spectrum Disorder
04/27
04/27
OTC-HOPE, NCT06255782: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Recruiting
1/2
13
Europe, US
ECUR-506
iECURE, Inc.
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
09/26
09/26
NCT05818943 / 2022-000317-14: Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder

Active, not recruiting
1
24
Europe, Canada, US, RoW
Radiprodil
GRIN Therapeutics, Inc., GRIN Therapeutics, Inc.
GRIN-related Disorders
11/26
11/26
Muntoni, Francesco
MIS51ON, NCT03992430 / 2018-001762-42: A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD)

Active, not recruiting
3
160
Europe, US, RoW
Eteplirsen, AVI-4658, EXONDYS 51, EXONDYS
Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
10/26
10/26
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
NCT03882827: Natural History of Duchenne Muscular Dystrophy

Recruiting
N/A
100
Europe
Genethon
Duchenne Muscular Dystrophy
06/23
06/23
SMA-REACH, NCT03520179: Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)

Recruiting
N/A
600
Europe
Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy
08/23
08/23
NCT04583917: Brain Involvement in Dystrophinopathies Part 1

Completed
N/A
377
Europe
Great Ormond Street Hospital for Children NHS Foundation Trust, Newcastle-upon-Tyne Hospitals NHS Trust, Leiden University Medical Center, Stichting Kempenhaeghe, Region Hovedstadens Apotek, Institut Necker Enfants Malades, Catholic University of the Sacred Heart, Università degli Studi di Ferrara, Universidad Complutense de Madrid
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
06/24
06/24
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-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
Ficicioglu, Can
NCT05099640 / 2021-000474-29: A Study of PTC923 in Participants With Phenylketonuria

Completed
3
157
Europe, Canada, US, RoW
PTC923, Placebo
PTC Therapeutics
Phenylketonuria
04/23
05/23
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting
3
43
Europe, Canada, Japan, US, RoW
Venglustat, imiglucerase, Cerezyme®
Sanofi, Sanofi-aventis recherche & développement
Gaucher's Disease Type III
09/25
10/26
SYNPHENY-3, NCT05764239: Efficacy and Safety of SYNB1934 in Patients With PKU

Terminated
3
35
Canada, US, RoW
SYNB1934v1, Placebo
Synlogic
Phenylketonuria
03/24
03/24
NCT05166161 / 2021-000497-28: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Recruiting
3
200
Europe, Canada, Japan, US, RoW
PTC923, Sepiapterin
PTC Therapeutics
Phenylketonuria
04/26
04/26
STARLIGHT, NCT04573023 / 2020-003200-14: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Recruiting
3
80
Europe, US, RoW
JR-141, Idursulfase, JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
01/26
01/26
NCT03566043: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Active, not recruiting
2/3
48
US, RoW
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
11/23
08/25
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Available
N/A
Europe, US, RoW
Idursulfase-IT, HGT-2310, TAK-609
Takeda
Hunter Syndrome
 
 
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
NCT04612764: Liver Disease in Urea Cycle Disorders

Recruiting
N/A
62
US
Baylor College of Medicine, Children's National Research Institute, Seattle Children's Hospital, Children's Hospital Colorado, Children's Hospital of Philadelphia
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
12/24
12/24
NCT00237315: Longitudinal Study of Urea Cycle Disorders

Recruiting
N/A
1500
Europe, Canada, US
Andrea Gropman, National Center for Research Resources (NCRR), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Rare Diseases Clinical Research Network
Brain Diseases, Metabolic, Inborn, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
07/25
07/26
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
N/A
40
Europe, Canada, US, RoW
University of Pennsylvania, Passage Bio, Inc.
GM1 Gangliosidosis
06/26
12/26
Giugliani, Roberto
PERIDOT, NCT05206773 / 2021-002350-90: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Active, not recruiting
3
122
Europe, Canada, Japan, US, RoW
Venglustat (GZ402671), Placebo
Sanofi, Sanofi-Aventis Recherche & Développement
Fabry Disease
12/25
12/26
STARLIGHT, NCT04573023 / 2020-003200-14: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Recruiting
3
80
Europe, US, RoW
JR-141, Idursulfase, JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
01/26
01/26
NCT03566043: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Active, not recruiting
2/3
48
US, RoW
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
11/23
08/25
NCT03632213: Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Active, not recruiting
2
10
RoW
Losartan, Placebo
Hospital de Clinicas de Porto Alegre, The Isaac Foundation
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidoses, MPS IV A, MPS VI, MPS - Mucopolysaccharidosis, Morquio A Syndrome, Morquio Syndrome A, Morquio Syndrome
05/23
08/23
RAINBOW, NCT05758922: Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Active, not recruiting
2
13
RoW
AZ-3102 (Dose 1), Placebo, AZ-3102 (Dose 2)
Azafaros A.G.
GM2 Gangliosidosis, Niemann-Pick Disease, Type C
03/24
12/24
NCT05737784: A Clinical Trial of PRAX-222 in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Recruiting
1/2
60
US, RoW
PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222 - Fixed Doses, Placebo
Praxis Precision Medicines
SCN2A-DEE, Epilepsy
08/25
09/27
PRONTO, NCT05109793: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study

Active, not recruiting
N/A
31
Europe, US, RoW
Azafaros A.G.
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
05/26
05/26
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
N/A
40
Europe, Canada, US, RoW
University of Pennsylvania, Passage Bio, Inc.
GM1 Gangliosidosis
06/26
12/26
Manzur, Adnan
NS, NCT06711692: The U.K. NorthStar Clinical Network

Not yet recruiting
N/A
300
Europe
University College, London, Muscular Dystrophy UK
Duchenne Muscular Dystrophy
04/25
06/25
McTague, Amy
NCT05818943 / 2022-000317-14: Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder

Active, not recruiting
1
24
Europe, Canada, US, RoW
Radiprodil
GRIN Therapeutics, Inc., GRIN Therapeutics, Inc.
GRIN-related Disorders
11/26
11/26
Gene-STEPS, NCT06082999: Shortening Time of Evaluation in Paediatric Epilepsy Services

Recruiting
N/A
300
Europe, Canada, US, RoW
Great Ormond Street Hospital for Children NHS Foundation Trust, Murdoch Childrens Research Institute, The Hospital for Sick Children, Boston Children's Hospital
Epilepsy
12/25
12/25

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