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 : September 28, 2018
|Condition or disease||Intervention/treatment||Phase|
|Crigler-Najjar Syndrome||Genetic: GNT0003||Phase 1 Phase 2|
|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 :||December 31, 2020|
|Estimated Study Completion Date :||December 31, 2020|
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: 29 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: 14 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: 29 months ]
- Scale 36-Item Short Form Survey (SF-36 Heath Survey for adults) - score range : 0 worse value : - better value (more favorable health state): 100. Scores represent the percentage of total possible score achieved. The scores from those questions that address each specific area of functional health status (STEP II chart) are then averaged together, for a final score within each of the 8 dimensions measured. (eg pain, physical functioning etc.). Outcome measure : change of quality of Life from baseline to Week 24 and week 48 (after IMP administration).
- Scale 10- Item Short Form Survey (SF-10 Heath Survey for children) - score range : 1 (worse value) to 6 (more favorable health state). : 100 The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. Outcome mesure : change of quality of Life from baseline to Week 24 and week 48 (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|