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 4 Products   0 Diseases  4 Products   8 Trials   823 News 
7 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Luxturna (voretigene neparvovec-rzyl) / Novartis, Roche
NCT00999609 / 2016-002109-20: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Checkmark AAPOS 2018
Apr 2018 - Apr 2018: AAPOS 2018
Checkmark For Leber Congenital Amaurosis
Jul 2017 - Jul 2017: For Leber Congenital Amaurosis
Checkmark
More
Active, not recruiting
3
31
US
AAV2-hRPE65v2,voretigene neparvovec-rzyl, AAV2-hRPE65v2, voretigene neparvovec-rzyl, gene therapy vector
Spark Therapeutics, Inc., Children's Hospital of Philadelphia, University of Iowa
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
07/15
01/30
CLTW888A11301, NCT04516369: Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Active, not recruiting
3
4
Japan
voretigene neparvovec
Novartis Pharmaceuticals
Biallelic RPE65 Mutation-associated Retinal Dystrophy
04/22
06/26
NCT01208389: Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Hourglass Jan 2017 - Dec 2017 : Anticipated 4-year durability data from 102 trial in RPE65-mediated IRDs
Checkmark Durability of effect after three years in P1 study 102 for RPE65-mediated IRDs at The Retina Society 2015
Oct 2015 - Oct 2015: Durability of effect after three years in P1 study 102 for RPE65-mediated IRDs at The Retina Society 2015
Active, not recruiting
1/2
12
US
voretigene neparvovec-rzyl, AAV2-hRPE65v2
Spark Therapeutics, Inc.
Leber Congenital Amaurosis
10/27
10/27
NCT03597399: A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Active, not recruiting
N/A
87
US
AAV2-hRPE65v2,voretigene neparvovec-rzyl, AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector
Spark Therapeutics, Inc.
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
06/25
06/25
NCT03602820: Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Active, not recruiting
N/A
41
NA
AAV2-hRPE65v2, Voretigene Neparvovec-rzyl
Spark Therapeutics, Inc.
Inherited Retinal Dystrophy Due to RPE65 Mutations
01/30
01/30
Hope (tetrahydrocannabinol/cannabidiol) / Zelira, The Children’s Hospital of Philadelphia
ACTRN12621000370808: Observational Study of two low dose medicinal cannabis oral oils to treat Autism associated behaviours.

Not yet recruiting
N/A
150
 
Zelira Therapeutics Ltd, Zelira Therapeutics Ltd
Autism Spectrum Disorder (ASD)
 
 
TmGPC2 01 CAR-T / Gilead
22CT012, NCT05650749: GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma

Recruiting
1
30
US
GPC2 CAR T cells
Stephan Grupp MD PhD, Children's Hospital of Philadelphia, Gilead Sciences, University of Pennsylvania, National Cancer Institute (NCI)
Refractory Neuroblastoma, Relapsed Neuroblastoma, High-risk Neuroblastoma
01/28
01/30
Undisclosed anti-FOXP3 ASO / AUM LifeTech
No trials found
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Luxturna (voretigene neparvovec-rzyl) / Novartis, Roche
NCT00999609 / 2016-002109-20: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Checkmark AAPOS 2018
Apr 2018 - Apr 2018: AAPOS 2018
Checkmark For Leber Congenital Amaurosis
Jul 2017 - Jul 2017: For Leber Congenital Amaurosis
Checkmark
More
Active, not recruiting
3
31
US
AAV2-hRPE65v2,voretigene neparvovec-rzyl, AAV2-hRPE65v2, voretigene neparvovec-rzyl, gene therapy vector
Spark Therapeutics, Inc., Children's Hospital of Philadelphia, University of Iowa
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
07/15
01/30
CLTW888A11301, NCT04516369: Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Active, not recruiting
3
4
Japan
voretigene neparvovec
Novartis Pharmaceuticals
Biallelic RPE65 Mutation-associated Retinal Dystrophy
04/22
06/26
NCT01208389: Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Hourglass Jan 2017 - Dec 2017 : Anticipated 4-year durability data from 102 trial in RPE65-mediated IRDs
Checkmark Durability of effect after three years in P1 study 102 for RPE65-mediated IRDs at The Retina Society 2015
Oct 2015 - Oct 2015: Durability of effect after three years in P1 study 102 for RPE65-mediated IRDs at The Retina Society 2015
Active, not recruiting
1/2
12
US
voretigene neparvovec-rzyl, AAV2-hRPE65v2
Spark Therapeutics, Inc.
Leber Congenital Amaurosis
10/27
10/27
NCT03597399: A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Active, not recruiting
N/A
87
US
AAV2-hRPE65v2,voretigene neparvovec-rzyl, AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector
Spark Therapeutics, Inc.
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
06/25
06/25
NCT03602820: Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Active, not recruiting
N/A
41
NA
AAV2-hRPE65v2, Voretigene Neparvovec-rzyl
Spark Therapeutics, Inc.
Inherited Retinal Dystrophy Due to RPE65 Mutations
01/30
01/30
Hope (tetrahydrocannabinol/cannabidiol) / Zelira, The Children’s Hospital of Philadelphia
ACTRN12621000370808: Observational Study of two low dose medicinal cannabis oral oils to treat Autism associated behaviours.

Not yet recruiting
N/A
150
 
Zelira Therapeutics Ltd, Zelira Therapeutics Ltd
Autism Spectrum Disorder (ASD)
 
 
TmGPC2 01 CAR-T / Gilead
22CT012, NCT05650749: GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma

Recruiting
1
30
US
GPC2 CAR T cells
Stephan Grupp MD PhD, Children's Hospital of Philadelphia, Gilead Sciences, University of Pennsylvania, National Cancer Institute (NCI)
Refractory Neuroblastoma, Relapsed Neuroblastoma, High-risk Neuroblastoma
01/28
01/30
Undisclosed anti-FOXP3 ASO / AUM LifeTech
No trials found

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