Gene Therapy for Severe Crigler Najjar Syndrome (CareCN)
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|ClinicalTrials.gov Identifier: NCT03466463|
Recruitment Status : Recruiting
First Posted : March 15, 2018
Last Update Posted : March 12, 2020
This is a Phase 1/2, multinational, open-label, escalating-dose study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):
- a follow-up of approximately 12 months (48 weeks)
- a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
|Condition or disease||Intervention/treatment||Phase|
|Crigler-Najjar Syndrome||Genetic: GNT0003||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||17 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy|
|Actual Study Start Date :||March 19, 2018|
|Estimated Primary Completion Date :||October 20, 2021|
|Estimated Study Completion Date :||April 27, 2026|
2 doses of the IMP assessed in a dose escalation, open-label, phase 1/2 study
Intravenous infusion, single dose
- Incidence of Adverse Events / Serious Adverse Events (Safety) : All Treatment Emergent Adverse Events, all Serious Adverse Events [ Time Frame: 12 months ]
Incidence of AE/SAE evaluated by changes in laboratory parameters, vital signs, physical examination, reported from baseline to each visit, evaluated for each dose and for the study as a whole. Clinically relevant abnormal findings on Laboratory values, Vital Signs, Physical findings will be reported as Adverse Events.
Incidence and Severity of Adverse Events for each body system will be presented for each dose level and summarized overall.
- Decrease of total Serum bilirubin level after interruption of daily phototherapy (Efficacy) [ Time Frame: 4 months ]Serum total bilirubin ≤ 300 µmol/L, 7 days after interruption of daily phototherapy occuring at week 16 after the administration of GNT0003.
- Quality of life assessment [ Time Frame: 60 months ]- Scale 36-Item Short Form Survey (SF-36 Health Survey for adults). Outcome measure: change of quality of life from Baseline to Week 24, Week 48 and yearly up to 48 months after IMP administration.
- Quality of life assessment [ Time Frame: 60 months ]- Scale 10-Item Short Form Survey (SF-10 Health Survey for children). Outcome measure: change of quality of life from Baseline to Week 24, Week 48 and yearly up to 48 months after IMP administration.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03466463
|Contact: Genethon Clinical Development Department||00 33 (0)1 69 47 10 firstname.lastname@example.org|
|Hopital Antoine BECLERE||Recruiting|
|Clamart, France, 92141|
|Contact 00 33 (0)1 45 37 42 72 email@example.com|
|Principal Investigator: Philippe LABRUNE, MD,PHD|
|ASST Papa Giovanni XXIII||Recruiting|
|Bergame, Italy, 24127|
|Contact 00390352674959 firstname.lastname@example.org|
|Principal Investigator: Lorenzo D'Antiga, MD, PHD|
|Azienda Ospedaliera Universitaria Federico II||Recruiting|
|Napoli, Italy, 80131|
|Contact 00390816132361 email@example.com|
|Principal Investigator: Nicola Brunetti-Pierri, MD, PHD|
|Amsterdam, Netherlands, 1105|
|Contact 0031-641476782 firstname.lastname@example.org|
|Principal Investigator: Ulrich Beuers, MD, PHD|
|Principal Investigator:||LABRUNE Philippe, Prof||Hopital Antoine Beclere|