The Royal Children's Hospital
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 5 Trials 
40 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Ryan, Monique
NCT02948244: Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD

Completed
N/A
11
RoW
Creatine Monohydrate, Placebo
Murdoch Childrens Research Institute
Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
01/21
07/22
Comi, Giacomo
TRAILBLAZER-ALZ 6, NCT05738486: A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer's Disease

Active, not recruiting
3
800
Europe, US
Donanemab, LY3002813, Placebo
Eli Lilly and Company
Alzheimer's Disease, Dementia, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders
05/24
05/25
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
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
NCT05973630: ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

Not yet recruiting
1/2
6
Europe, US
ATA-200
Atamyo Therapeutics
LGMD2C
07/26
01/32
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
Perrett, Kirsten P
MIST+, NCT05382494: Intranasal Steroid as Medical Therapy For Sleep-Disordered Breathing in Children

Active, not recruiting
4
130
RoW
Mometasone Furoate 50mcg Nasal Spray, Sensease Nasal Allergy Relief Nasal Spray, Sodium Chloride 0.9 % Nasal Spray, Intranasal Saline Spray
Murdoch Childrens Research Institute, Monash Health, Royal Children's Hospital
Sleep Disorder; Breathing-Related, Snoring, Obstructive Sleep Apnea of Child, Sleep Disorders in Children, Tonsillar Hypertrophy, Adenoidal Disorder
12/25
12/25
VITALITY, NCT02112734: Can Vitamin D Supplementation in the First Year of Life Prevent Food Allergy in Infants? the Trial: Parts 1&2

Active, not recruiting
4
2739
RoW
Vitamin D, cholecalciferol, placebo, placebo is identical carrier minus vitamin D
Murdoch Childrens Research Institute
Food Allergy
04/28
12/28
TreEat, NCT04801823: The Study- Can Early Introduction of Tree Nuts Prevent Tree Nut Allergy in Infants with Peanut Allergy

Active, not recruiting
3
212
RoW
Home introduction, Multi Nut oral food challenge
Murdoch Childrens Research Institute
Food Allergy in Infants, Nut Allergy
11/25
11/25
adored, NCT05003804: Allergic Disease Onset Prevention Study

Active, not recruiting
1/2
264
US, RoW
STMC-103H, Placebo
Siolta Therapeutics, Inc.
Atopic Dermatitis, Type 1 Hypersensitivity
11/24
10/25
NCT06273605: The Egg Up Trial - Testing a New Treatment Pathway for Infants With Newly Diagnosed Egg Allergy

Active, not recruiting
N/A
100
RoW
Egg Ladder
Murdoch Childrens Research Institute
Food Allergy, Food Allergy in Infants
08/25
08/25
LMNOP, NCT05049512: Low Dose Multi-Nut Oral Immunotherapy in Pre-schoolers With a Multi-Nut Allergy

Recruiting
N/A
45
RoW
Multi-nut OIT
Murdoch Childrens Research Institute
Allergy, Nut
12/25
12/25
ARISE, NCT06686446: Allergic Rhinitis Improvement Through Strategic Education: The Trial

Not yet recruiting
N/A
194
RoW
Enhanced Education Package
Murdoch Childrens Research Institute
Allergy
09/25
05/26
Osorio, Andres Nascimento
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
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
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
Toro, Claudia
NCT05615376: Beat to Beat A Study in Paediatric, Adolescent and Young Adult Patients Who Are Undergoing or Have Undergone Cancer Therapy to Evaluate the Agreement Between QTc Measured Using 12 Lead Electrocardiogram (ECG) and a Wearable Device ECG

Active, not recruiting
N/A
46
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
06/24
12/24
Luen, Stephen
IMPARP-HRD, NCT04985721: A Trial of Pamiparib With Tislelizumab in Patients With Advanced Tumours With Homologous Recombination Repair Defects

Recruiting
2
60
RoW
Pamiparib, Tislelizumab
Peter MacCallum Cancer Centre, Australia
Cancer
08/25
08/25
BELLA, NCT04739670: Evaluating the Efficacy and Safety of Bevacizumab, Carboplatin, Gemcitabine and Atezolizumab in Breast Cancer

Recruiting
2
31
RoW
Atezolizumab, Bevacizumab, Gemcitabine, Carboplatin
Peter MacCallum Cancer Centre, Australia
Metastatic Triple Negative Breast Cancer
09/25
09/25
TARGET-VM, NCT05983159: A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

Recruiting
2
50
RoW
Alpelisib, BYL719, Mirdametinib
Murdoch Childrens Research Institute, Peter MacCallum Cancer Centre, Australia, Royal Children's Hospital
Slow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous Malformation, Lymphatic Malformation, Low Flow, Lymphatic Malformation, Lymphangioma, Arteriovenous Malformations, Venous Malformation, Low Flow, Cystic Hygroma, Vascular Anomaly, Vascular Anomalies, PI3K Gene Mutation, MAP2K1 Gene Mutation, PIK3CA-related Overgrowth Spectrum, Arteriovenous Malformation (AVM), KRAS G12C, KRAS G12D
08/26
12/26
KEYNOTE-F49, NCT05763004: A Clinical Study to Evaluate the Safety, Tolerability and Efficacy of IOS-1002 Administered Alone and in Combination with Pembrolizumab, a PD-1 Monoclonal Antibody in Advanced Solid Tumors

Recruiting
1
140
RoW
IOS-1002, IOS-1002 + KEYTRUDA® (pembrolizumab)
ImmunOs Therapeutics AG, Merck Sharp & Dohme LLC
Solid Tumor, Adult
05/25
05/25
TAILOR, NCT04801966: Safety and Oversight of the Individually Tailored Treatment Approach: A Novel Pilot Study

Terminated
N/A
3
RoW
Trametinib, Cobimetinib, Binimetinib, Alpelisib, Vemurafenib, Dabrafenib, Encorafenib, Palbociclib, Olaparib, Ribociclib, Abemaciclib, Talazoparib, Nivolumab, Atezolizumab, Pembrolizumab
Peter MacCallum Cancer Centre, Australia
Cancer
12/22
12/22
NCT05653388: Metastatic Leiomyosarcoma Biomarker Protocol

Recruiting
N/A
200
US, RoW
Plasma Collection
University of Michigan Rogel Cancer Center, National Cancer Institute (NCI)
Leiomyosarcoma
12/26
12/26
Conyers, Assoc Professor R
NCT05401656: A Study to Validate the Apple Watch's ECG Application in Children During or After Cancer Therapy

Not yet recruiting
N/A
40
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
11/22
05/23
Conyers, Associate Professor R
NCT05401656: A Study to Validate the Apple Watch's ECG Application in Children During or After Cancer Therapy

Not yet recruiting
N/A
40
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
11/22
05/23
Toro, Doctor C
NCT05401656: A Study to Validate the Apple Watch's ECG Application in Children During or After Cancer Therapy

Not yet recruiting
N/A
40
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
11/22
05/23
Felmingham, Ben
NCT05401656: A Study to Validate the Apple Watch's ECG Application in Children During or After Cancer Therapy

Not yet recruiting
N/A
40
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
11/22
05/23
Conyers, A/Prof Rachel
BRUNO-PIC, NCT06529536: Utilising Genotype Informed Bayesian Dosing of Tacrolimus in Children Post Solid Organ Transplantation.

Recruiting
2
45
RoW
Genotyping for CYP3A4 and CYP3A5 genes, Use of NextDose platform, Tacrolimus, Prograf (Brand)
Murdoch Childrens Research Institute
Solid Organ Transplant
08/27
08/27
NCT05615376: Beat to Beat A Study in Paediatric, Adolescent and Young Adult Patients Who Are Undergoing or Have Undergone Cancer Therapy to Evaluate the Agreement Between QTc Measured Using 12 Lead Electrocardiogram (ECG) and a Wearable Device ECG

Active, not recruiting
N/A
46
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
06/24
12/24
MARVEL-PIC, NCT05667766: Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children

Recruiting
N/A
880
RoW
Release of Extended Pharmacogenomics Report at Week 1, Release of Extended Pharmacogenomics Report at Week 13
Murdoch Childrens Research Institute
Neoplasms, Bone Marrow Transplantation
08/25
08/26
Collier, Lane
NCT05615376: Beat to Beat A Study in Paediatric, Adolescent and Young Adult Patients Who Are Undergoing or Have Undergone Cancer Therapy to Evaluate the Agreement Between QTc Measured Using 12 Lead Electrocardiogram (ECG) and a Wearable Device ECG

Active, not recruiting
N/A
46
RoW
ECG application, 12 lead ECG
Murdoch Childrens Research Institute
Neoplasms, Cardiac Arrhythmia
06/24
12/24
Danchin, Margie
NCT05613751: Enhancing Protection Against Influenza and COVID-19 for Pregnant Women and Medically at Risk Children

Recruiting
N/A
1038
RoW
Nudge
University of Adelaide
Influenza, COVID-19
06/24
12/24
Murphy, Kate
IAA, NCT05742243: Insulin and Abatacept in Recently-diagnosed Type 1 Diabetes

Active, not recruiting
2
62
RoW
Abatacept (CTLA4-Ig) and nasal insulin (Humulin R®), Abatacept and nasal insulin, Abatacept (CTLA4-Ig) and nasal placebo (0.9% sodium chloride), Abatacept and nasal placebo
Melbourne Health, National Health and Medical Research Council, Australia, Juvenile Diabetes Research Foundation
Type 1 Diabetes, Diabetes Mellitus, Type 1
02/26
02/27
Penington, Tony
TARGET-VM, NCT05983159: A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

Recruiting
2
50
RoW
Alpelisib, BYL719, Mirdametinib
Murdoch Childrens Research Institute, Peter MacCallum Cancer Centre, Australia, Royal Children's Hospital
Slow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous Malformation, Lymphatic Malformation, Low Flow, Lymphatic Malformation, Lymphangioma, Arteriovenous Malformations, Venous Malformation, Low Flow, Cystic Hygroma, Vascular Anomaly, Vascular Anomalies, PI3K Gene Mutation, MAP2K1 Gene Mutation, PIK3CA-related Overgrowth Spectrum, Arteriovenous Malformation (AVM), KRAS G12C, KRAS G12D
08/26
12/26
Pathmanathan, Lydia
TARGET-VM, NCT05983159: A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

Recruiting
2
50
RoW
Alpelisib, BYL719, Mirdametinib
Murdoch Childrens Research Institute, Peter MacCallum Cancer Centre, Australia, Royal Children's Hospital
Slow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous Malformation, Lymphatic Malformation, Low Flow, Lymphatic Malformation, Lymphangioma, Arteriovenous Malformations, Venous Malformation, Low Flow, Cystic Hygroma, Vascular Anomaly, Vascular Anomalies, PI3K Gene Mutation, MAP2K1 Gene Mutation, PIK3CA-related Overgrowth Spectrum, Arteriovenous Malformation (AVM), KRAS G12C, KRAS G12D
08/26
12/26
Penington, Anthony
TARGET-VM, NCT05983159: A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

Recruiting
2
50
RoW
Alpelisib, BYL719, Mirdametinib
Murdoch Childrens Research Institute, Peter MacCallum Cancer Centre, Australia, Royal Children's Hospital
Slow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous Malformation, Lymphatic Malformation, Low Flow, Lymphatic Malformation, Lymphangioma, Arteriovenous Malformations, Venous Malformation, Low Flow, Cystic Hygroma, Vascular Anomaly, Vascular Anomalies, PI3K Gene Mutation, MAP2K1 Gene Mutation, PIK3CA-related Overgrowth Spectrum, Arteriovenous Malformation (AVM), KRAS G12C, KRAS G12D
08/26
12/26
Simatos, Dimi
NCT05613751: Enhancing Protection Against Influenza and COVID-19 for Pregnant Women and Medically at Risk Children

Recruiting
N/A
1038
RoW
Nudge
University of Adelaide
Influenza, COVID-19
06/24
12/24
Jessop, Sophie
MARVEL-PIC, NCT05667766: Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children

Recruiting
N/A
880
RoW
Release of Extended Pharmacogenomics Report at Week 1, Release of Extended Pharmacogenomics Report at Week 13
Murdoch Childrens Research Institute
Neoplasms, Bone Marrow Transplantation
08/25
08/26
Magri, Francesa
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

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