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 28, 2023
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This is a Phase 1/2, multinational, open-label, 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 |
Allocation: | N/A |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Other |
Official Title: | A Phase I/II, Open Label, 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 : | March 30, 2026 |
Estimated Study Completion Date : | March 30, 2030 |
Arm | Intervention/treatment |
---|---|
Experimental: GNT0003
2 doses of the IMP assessed in the dose escalation, open-label, phase 1/2 study
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Genetic: GNT0003
Intravenous infusion, single dose |
- Proportion of patients having received the selected dose of GNT0003 with serum bilirubin ≤ 300µmol/L within 48 meeks after GNT0003 administration and without phototherapy from week 16 [ Time Frame: 48 weeks ]Decrease of total Serum bilirubin level after interruption of daily phototherapy (Efficacy); change in serum total biliirubin from baseline to week 48
- Incidence of Treatment Emergent Adverse Events or Treatement Serious Adverse Events [ Time Frame: 48 weeks ]
Incidence of AE/SAE evaluated by changes in laboratory parameters, vital signs, physical examination, reported from baseline to each visit study. 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.
- Change in Health-related quality of Life for Adults from Baseline to Week 48 after GNT0003 administration [ Time Frame: 48 weeks ]
Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration.
Scale 36-Item Short Form Survey (SF-36 Health Survey) for adults: from 0 (negative to health) to 100 (positive to health)
- Change in Health-related quality of Life for Children from Baseline to Week 48 after GNT0003 administration [ Time Frame: 48 weeks ]
Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration.
PedsQL 4.0 Generic Core Scale for pediatrics: 0-100 scale where higher scores indicate better health-related quality of life
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Ages Eligible for Study: | 9 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy
- Male or female at least 9 years at the date of signature of informed consent
- Patient able to give informed assent and/or consent in writing
Exclusion Criteria:
- Patients who underwent liver transplantation
- Patients with chronic hepatitis B or C
- Patients infected with Human immunodeficiency virus (HIV)
- Patients with significant underlying liver disease
- Patients with significant encephalopathy
- Participation in any other investigational trial during this trial
- Patients unable or unwilling to comply with the protocol requirements
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 32 | clinical_development@genethon.fr |
France | |
Hopital Antoine BECLERE | Recruiting |
Clamart, France, 92141 | |
Contact 00 33 (0)1 45 37 42 72 philippe.labrune@abc.aphp.fr | |
Principal Investigator: Philippe LABRUNE, MD,PHD | |
Italy | |
ASST Papa Giovanni XXIII | Recruiting |
Bergame, Italy, 24127 | |
Contact 00390352674959 ldantiga@asst-pg23.it | |
Principal Investigator: Lorenzo D'Antiga, MD, PHD | |
Azienda Ospedaliera Universitaria Federico II | Recruiting |
Napoli, Italy, 80131 | |
Contact 00390816132361 brunetti@tigem.it | |
Principal Investigator: Nicola Brunetti-Pierri, MD, PHD | |
Netherlands | |
AMC | Recruiting |
Amsterdam, Netherlands, 1105 | |
Contact 0031-641476782 u.h.beuers@amc.uva.nl | |
Principal Investigator: Ulrich Beuers, MD, PHD |
Principal Investigator: | LABRUNE Philippe, Prof | Hopital Antoine Beclere |
Responsible Party: | Genethon |
ClinicalTrials.gov Identifier: | NCT03466463 |
Other Study ID Numbers: |
GNT-012-CRIG |
First Posted: | March 15, 2018 Key Record Dates |
Last Update Posted: | March 28, 2023 |
Last Verified: | February 2023 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Crigler-Najjar Adeno Associated Virus |
Cardiomyopathies Crigler-Najjar Syndrome Syndrome Disease Pathologic Processes Hyperbilirubinemia, Hereditary |
Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases Heart Diseases Cardiovascular Diseases |