Crigler Najjar Syndrome gene therapy trials

CRIGLER-NAJJAR
SYNDROME

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 - CureCN

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
December 2018: The first patient affected by Crigler-Najjar syndrome has been administered with the gene therapy vector in CURE CN trial sponsored by Genethon. A second patient has been subsequently treated. No significant side effectes have been reported, but the descrease in hyperbilirubinemia observed in both patients was transient.
LAST UPDATE: November 2019: The first Italian patient with Crigler-Najjar type I syndrome underwent gene therapy treatment at the Papa Giovanni XXIII Hospital in Bergamo by Prof. Lorenzo D'Antiga's team as part of the clinical trial CureCN. The dose of vector used in this patient was higher than in the previuously treated ones.

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.