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Genethon
Sponsor

Genethon

France 1 Rue De L Internationale, 91000 Evry-Courcouronnes, France

About the sponsor

Clinical research includes Crigler-Najjar syndrome and severe Crigler-Najjar syndrome in patients requiring phototherapy, with a focus on restoring bilirubin metabolism and addressing persistent hyperbilirubinemia.

  • Crigler-Najjar syndrome
  • Severe Crigler-Najjar syndrome
  • Phototherapy-dependent disease

Work in this area centers on liver-directed gene therapy and long-term clinical assessment of metabolic correction.

About Specializations

Genethon runs clinical trials in conditions such as Crigler-Najjar syndrome, Wiskott-Aldrich syndrome, Duchenne muscular dystrophy, Chronic granulomatous disease, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
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Type of trial
Recruitment status
Quick filters

5 clinical trials

Crigler-Najjar syndrome

Recruiting

Study of GNT0003 and imlifidase in adults with Crigler-Najjar syndrome who require daily phototherapy and have pre-existing AAV8 antibodies

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Amoxicillin Sodium
  • Sponsor:Genethon

Wiskott-Aldrich syndrome

Recruiting

Long-Term Safety Study of Gene Therapy with Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Etuvetidigene Autotemcel
  • Sponsor:Genethon

Crigler-Najjar syndrome

Recruiting

Study on Gene Therapy GNT0003 for Patients with Severe Crigler-Najjar Syndrome Requiring Phototherapy

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Abiraterone Acetate
  • Sponsor:Genethon

Duchenne muscular dystrophy

Recruiting

Study of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk

Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Eculizumab
  • Sponsor:Genethon

Chronic granulomatous disease

Not recruiting

Study of G1XCGD Lentiviral Vector in Patients with X-Linked Chronic Granulomatous Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Cells Transduced With The G1Xcgd Lentiviral Vector Containing The Human Cybb Gene
  • Sponsor:Genethon

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