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

   Remove FilterRemove Filter

 
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
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
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
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
Vincent, Ajoy
No trials found

Download Options