University of Toronto, Hospital for Sick Children
Welcome,         Profile    Billing    Logout  
 2 Trials 
13 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Heon, Elise
NCT04671433 / 2020-002873-88: Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Completed
3
97
Europe, Canada, US, RoW
Genetic: AAV5-hRKp.RPGR, botaretigene sparoparvovec
Janssen Research & Development, LLC, Janssen Research & Development, LLC
X-Linked Retinitis Pigmentosa
09/24
09/24
MGT-RPGR-022, NCT04794101 / 2020-002255-37: Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Active, not recruiting
3
97
Europe, Canada, US, RoW
Genetic: AAV5-hRKp.RPGR Intermediate Dose, botaretigene sparoparvovec, Genetic: AAV5-hRKp.RPGR Low Dose
Janssen Research & Development, LLC, Janssen Research & Development, LLC
X-Linked Retinitis Pigmentosa
09/29
12/29
NCT00110110: Combination Chemotherapy and Cyclosporine Followed by Focal Therapy for Bilateral Retinoblastoma

Active, not recruiting
2
71
Canada, RoW
filgrastim, granulocyte colony-stimulating factor (G-CSF), Carboplatin, Cyclosporine, Etoposide, vincristine sulfate, vincristine, cryosurgery, cryotherapy, laser therapy
The Hospital for Sick Children, Terry Fox Foundation
Retinoblastoma
04/16
12/24
NCT03349242: Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

Completed
N/A
140
Europe, Canada, US
MeiraGTx UK II Ltd, Janssen, LP
Retinitis Pigmentosa
04/24
04/24
Uni-Rare, NCT05589714: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

Recruiting
N/A
1500
Europe, Canada, US, RoW
Jaeb Center for Health Research, Foundation Fighting Blindness
Inherited Retinal Degeneration, Retinitis Pigmentosa
12/28
12/28
Sallum, Juliana M
BRIGHTEN, NCT04855045 / 2020-000535-45: An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

Recruiting
2/3
15
Europe, Canada, RoW
sepofarsen, QR-110
ProQR Therapeutics
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
12/23
12/23
Uni-Rare, NCT05589714: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

Recruiting
N/A
1500
Europe, Canada, US, RoW
Jaeb Center for Health Research, Foundation Fighting Blindness
Inherited Retinal Degeneration, Retinitis Pigmentosa
12/28
12/28
Porto, Fernanda
BRIGHTEN, NCT04855045 / 2020-000535-45: An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

Recruiting
2/3
15
Europe, Canada, RoW
sepofarsen, QR-110
ProQR Therapeutics
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
12/23
12/23
Uni-Rare, NCT05589714: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

Recruiting
N/A
1500
Europe, Canada, US, RoW
Jaeb Center for Health Research, Foundation Fighting Blindness
Inherited Retinal Degeneration, Retinitis Pigmentosa
12/28
12/28
GYROS, NCT05312736: Gyrate Atrophy Ocular and Systemic Study

Recruiting
N/A
45
Europe, Canada, US, RoW
Jaeb Center for Health Research, Conquering Gyrate Atrophy Foundation, Foundation Fighting Blindness
Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy, Ornithine-δ-aminotransferase, OAT, Chorioretinal Degeneration
12/28
06/29
Vincent, Ajoy
RUSH1F, NCT04765345: Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

Active, not recruiting
N/A
40
Europe, Canada, US, RoW
Jaeb Center for Health Research, Usher 1F Collaborative, Marjorie C. Adams Foundation, Foundation Fighting Blindness
Retinal Degeneration, Retinitis Pigmentosa, Eye Diseases, Hereditary
06/27
06/27
GYROS, NCT05312736: Gyrate Atrophy Ocular and Systemic Study

Recruiting
N/A
45
Europe, Canada, US, RoW
Jaeb Center for Health Research, Conquering Gyrate Atrophy Foundation, Foundation Fighting Blindness
Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy, Ornithine-δ-aminotransferase, OAT, Chorioretinal Degeneration
12/28
06/29
NCT05386134: Adaptive Optics Retinal Imaging in Inherited and Acquired Retinal Disorders

Recruiting
N/A
200
Canada
Adaptive Optics Retinal Camera
The Hospital for Sick Children
Genetic Disease, Inherited Disease
06/32
06/33

Download Options