The University of British Columbia, Children's and Womens Health Centre of BC Branch
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 2 Trials 
6 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Selby, Kathryn
ULYSSES, NCT05933057: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Recruiting
3
138
Europe, Canada
Givinostat, ITF2357, Placebo
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
02/28
02/28
NCT04833894 / 2020-005841-18: Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis

Recruiting
2/3
12
Europe, Canada, US, RoW
Efgartigimod IV
argenx
Generalized Myasthenia Gravis
03/27
03/27
NCT05185622: A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Completed
2
54
Canada
Vamorolone, VBP15
Santhera Pharmaceuticals, Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
07/24
07/24
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
NCT03863119: Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Approved for marketing
N/A
US
Vamorolone
Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
 
 
INFORM SMA, NCT06396325: A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy

Recruiting
N/A
22
Canada
Tales from the Magic Keep™, Usual Care
Maryam Oskoui, MD, MSc, FRCPC, FAAN
Spinal Muscular Atrophy
12/26
12/27
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Selby, Kathryn
ULYSSES, NCT05933057: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Recruiting
3
138
Europe, Canada
Givinostat, ITF2357, Placebo
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
02/28
02/28
NCT04833894 / 2020-005841-18: Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis

Recruiting
2/3
12
Europe, Canada, US, RoW
Efgartigimod IV
argenx
Generalized Myasthenia Gravis
03/27
03/27
NCT05185622: A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Completed
2
54
Canada
Vamorolone, VBP15
Santhera Pharmaceuticals, Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
07/24
07/24
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
NCT03863119: Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Approved for marketing
N/A
US
Vamorolone
Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
 
 
INFORM SMA, NCT06396325: A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy

Recruiting
N/A
22
Canada
Tales from the Magic Keep™, Usual Care
Maryam Oskoui, MD, MSc, FRCPC, FAAN
Spinal Muscular Atrophy
12/26
12/27

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