CRIGLER-NAJJAR 
SYNDROME


 

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 CLINICAL TRIALS on CRIGLER-NAJJAR SYNDROME:

Several gene therapy clinical trials are being considered as a therapeutic option for the treatment of Crigler-Najjar patients:

 

    • Title: A phase I/II, open label, escalating dose study to evaluate safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy.

      Abbreviated trial name: CareCN

      Purpose: To assess safety, tolerability and efficacy of a single intravenous administration of Adeno-associated Viral Vector expressing the UGT1A1 transgene in patients with severe Crigler-Najjar syndrome requiring phototherapy.

      Sponsor: Genethon - Developer: Genethon and Spark Therapeutics

      Sponsor:This project has received funds from the European 's Union Horizon 2020 research and innovation programme under the grant agreement number 755225 Cure CN Horizon 2020 Project

      EU Clinical Trial Register number: 2017-000506-37

      EU Clinical Trial Link: https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-000506-37/NL

      ClinicalTrials.gov Identifier: NCT03466463

      ClinicalTrial.gov Link: https://clinicaltrials.gov/ct2/show/NCT03466463?cond=Crigler-Najjar+Syndrome&rank=1

       

       

    • Title: Clinical Assessment Study in Crigler-Najjar Syndrome

      Abbreviated trial name: LUSTRO

      ClinicalTrials.gov Identifier: NCT03078881

      Purpose: Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.

      Sponsor: Audentes Therapeutics

      ClinicalTrials.gov Identifier: NCT03078881

      ClinicalTrials.gov Link: https://clinicaltrials.gov/ct2/show/NCT03078881

        

       

    • Title:  Gene Transfer Clinical Study in Crigler-Najjar Syndrome (VALENS)

      Abbreviated trial name: VALENS

      Purpose: Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of adeno-associated viral vector expressing the UGT1A1 transgene in subjects with Crigler-Najjar aged ≥1 year.

      Sponsor: Audentes Therapeutics

      ClinicalTrials.gov Identifier: NCT03223194

      ClinicalTrials.gov Link: https://clinicaltrials.gov/ct2/show/NCT03223194

    • LAST UPDATE: February 2018: First patient affected by Crigler-Najjar syndrome administered with gene therapy vector in VALENS clinical trial sponsored by Audentes.

 

 

 

  

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