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 4 Products   2 Diseases  4 Products   4 Trials   35 News 
6 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
autologous CD34+ cells transduced with WASP lentiviral vector / Genethon, Boston Children's Hospital
WAS FUP, NCT02333760 / 2014-000274-20: Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Active, not recruiting
1/2
10
Europe
Autologous CD34+ cells transduced with WASP lentiviral vector
Genethon
Wiskott-Aldrich Syndrome
10/32
10/32
OTL-102 / Genethon, Kyowa Kirin
2014-002222-12: Gene therapy with autologous genetically-modified CD34+ cells for X-Linked Chronic Granulomatous Disease (X-CGD)

Not yet recruiting
1/2
5
Europe
Autologous CD34+ cells transduced with the G1XCGD lentiviral vector, Suspension for injection
Genethon, Genethon, Net4CGD project
Chronic Granulomatous Disease (CGD) is a rare inherited disorder (1/250,000) of the phagocytes characterized by the inability of phagocytes (monocytes and neutrophils) to produce reactive oxygen species (ROS) in response to a stimulus, due to the absence of NADPH oxidase activity. Affected patients present an elevated susceptibility to bacterial and fungal infections, as well as an excessive inflammatory response that leads to granuloma formation., Chronic Granulomatous Disease (CGD) is a genetic disorder that affects boys. It is caused by errors in a gene that makes part of the immune system., Diseases [C] - Immune System Diseases [C20]
 
 
NCT02234934: Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Completed
1/2
10
US
Lentiviral G1XCGD Gene Therapy, G1XCGD (pCCLChimGp91/VSVg lentiviral vector)
University of California, Los Angeles, Boston Children's Hospital, National Heart, Lung, and Blood Institute (NHLBI), Genethon, California Institute for Regenerative Medicine (CIRM)
Granulomatous Disease, Chronic, X-linked
09/24
12/24
G1XCGD.01, NCT01855685 / 2012-000242-35: Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)

Active, not recruiting
1/2
3
Europe
X vivo gene therapy
Genethon
X-Linked Chronic Granulomatous Disease
09/32
09/32
NCT02757911 / 2014-002222-12: Gene Therapy for X-linked Chronic Granulomatous Disease

Active, not recruiting
1/2
3
Europe
X vivo gene therapy
Genethon, Genethon, Net4CGD project
X-Linked Chronic Granulomatous Disease
06/34
06/34
GNT 0004 / Sarepta Therap
2020-002093-27: A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).

Not yet recruiting
2/3
51
Europe
rAAV8-hMD1, GNT0004, Concentrate for solution for infusion
Genethon, Genethon
Duchenne Muscular Dystrophy, Duchenne's disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
autologous CD34+ cells transduced with WASP lentiviral vector / Genethon, Boston Children's Hospital
WAS FUP, NCT02333760 / 2014-000274-20: Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Active, not recruiting
1/2
10
Europe
Autologous CD34+ cells transduced with WASP lentiviral vector
Genethon
Wiskott-Aldrich Syndrome
10/32
10/32
OTL-102 / Genethon, Kyowa Kirin
2014-002222-12: Gene therapy with autologous genetically-modified CD34+ cells for X-Linked Chronic Granulomatous Disease (X-CGD)

Not yet recruiting
1/2
5
Europe
Autologous CD34+ cells transduced with the G1XCGD lentiviral vector, Suspension for injection
Genethon, Genethon, Net4CGD project
Chronic Granulomatous Disease (CGD) is a rare inherited disorder (1/250,000) of the phagocytes characterized by the inability of phagocytes (monocytes and neutrophils) to produce reactive oxygen species (ROS) in response to a stimulus, due to the absence of NADPH oxidase activity. Affected patients present an elevated susceptibility to bacterial and fungal infections, as well as an excessive inflammatory response that leads to granuloma formation., Chronic Granulomatous Disease (CGD) is a genetic disorder that affects boys. It is caused by errors in a gene that makes part of the immune system., Diseases [C] - Immune System Diseases [C20]
 
 
NCT02234934: Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Completed
1/2
10
US
Lentiviral G1XCGD Gene Therapy, G1XCGD (pCCLChimGp91/VSVg lentiviral vector)
University of California, Los Angeles, Boston Children's Hospital, National Heart, Lung, and Blood Institute (NHLBI), Genethon, California Institute for Regenerative Medicine (CIRM)
Granulomatous Disease, Chronic, X-linked
09/24
12/24
G1XCGD.01, NCT01855685 / 2012-000242-35: Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)

Active, not recruiting
1/2
3
Europe
X vivo gene therapy
Genethon
X-Linked Chronic Granulomatous Disease
09/32
09/32
NCT02757911 / 2014-002222-12: Gene Therapy for X-linked Chronic Granulomatous Disease

Active, not recruiting
1/2
3
Europe
X vivo gene therapy
Genethon, Genethon, Net4CGD project
X-Linked Chronic Granulomatous Disease
06/34
06/34
GNT 0004 / Sarepta Therap
2020-002093-27: A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).

Not yet recruiting
2/3
51
Europe
rAAV8-hMD1, GNT0004, Concentrate for solution for infusion
Genethon, Genethon
Duchenne Muscular Dystrophy, Duchenne's disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 

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