6 completed, 3 recruiting, 3 terminated, 1 unknown. 4 Phase 1, 3 Not applicable, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Terminated 2, Completed 1, Recruiting 1 |
| Not applicable | 3 | Completed 1, Recruiting 1, Unknown 1 |
| Phase 2 | 3 | Recruiting 2, Completed 1 |
Follow Crigler-Najjar Syndrome: new trials by email Literature Review of Crigler-Najjar Syndrome trials Research Report on Crigler-Najjar Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome Imlifidase, GNT0003 - Crigler-Najjar Syndrome | Phase 2 | Recruiting | Genethon | 3 | 2024-11-08 | 2024-12-03 |
| Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN) GT-UGT1A1-AAV8-02 - Crigler-Najjar Syndrome Type I | Phase 1 / Phase 2 | Recruiting | Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare | 5 | 2024-11-06 | 2025-01-13 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| HepaStem Long-Term Safety Registry Urea Cycle Disorder, Crigler-Najjar Syndrome | Terminated | Cellaion SA | 44 | 2018-04-04 | 2024-05-29 | |
| Gene Therapy for Severe Crigler Najjar Syndrome GNT0003 - Crigler-Najjar Syndrome | Not applicable | Recruiting | Genethon | 17 | 2018-03-19 | 2023-03-28 |
| Gene Transfer Clinical Study in Crigler-Najjar Syndrome AT342 - Crigler-Najjar Syndrome | Phase 1 | Terminated | Audentes Therapeutics | 1 | 2017-09-08 | 2022-05-18 |
| Clinical Assessment Study in Crigler-Najjar Syndrome Crigler-Najjar Syndrome | Completed | Audentes Therapeutics | 8 | 2017-03-10 | 2019-02-06 | |
| New Phototherapy Device to Treat Patients With Crigler-Najjar Disease Arm1 homemade phototherapy treatment, DRAP - Crigler Najjar Syndrome, Children | Not applicable | Unknown | Assistance Publique - Hôpitaux de Paris | 6 | 2014-12-01 | 2017-09-12 |
| Immunity Against AAV in Crigler Najjar Patient Blood sample for immunology - Crigler Najjar Syndrome | Completed | Genethon | 49 | 2014-11-01 | 2017-12-13 | |
| Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001) Urea Cycle Disorders, Crigler Najjar Syndrome | Completed | Cellaion SA | 9 | 2013-03-01 | 2019-05-28 | |
| Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) HepaStem - Urea Cycle Disorders, Crigler Najjar Syndrome | Phase 1 / Phase 2 | Completed | Cellaion SA | 20 | 2012-03-01 | 2020-10-19 |
| Hepatocyte Transplantation for Liver Based Metabolic Disorders human hepatocyte transplantation, Preparative Radiation Therapy - Metabolic Diseases | Phase 1 | Terminated | Ira Fox | 5 | 2011-03-01 | 2022-12-07 |
| Orlistat Treatment of Crigler-Najjar Disease orlistat - Crigler-Najjar Syndrome | Not applicable | Completed | University Medical Center Groningen | 16 | 2003-09-01 | 2007-04-18 |
Urea Cycle Disorders, Inborn (4) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Citrullinemia (2) · Ornithine Carbamoyltransferase Deficiency Disease (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1) · Acidemia, isovaleric (1) · Acute-On-Chronic Liver Failure (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Adrenal Hyperplasia, Congenital (1) · Adrenoleukodystrophy (1) · Andersen Syndrome (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Genetic Diseases, Inborn (13) · Hyperbilirubinemia, Hereditary (13) · Metabolism, Inborn Errors (13) · Nutritional and Metabolic Diseases (13) · Metabolic Diseases (12) · Amino Acid Metabolism, Inborn Errors (5) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Nervous System Diseases (5) · Digestive System Diseases (2) · Disease Attributes (2) · Genetic Diseases, X-Linked (2)
Blood Specimen Collection (1) · Mac-1-like protein, Streptococcus (1) · Orlistat (1)
Cellaion SA (3) · Genethon (3) · Audentes Therapeutics (2) · Assistance Publique - Hôpitaux de Paris (1) · Centre Hospitalier Universitaire de Liege (1) · Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (1) · Ira Fox (1) · University Medical Center Groningen (1)
France (6) · Belgium (4) · Israel (3) · United Kingdom (3) · United States (3) · Italy (2) · Netherlands (2) · Bulgaria (1) · Poland (1) · Russia (1) · Spain (1)
Anja M. Hafkamp, MD (1) · Dries Dobbelaere, MD/Prof (1) · Giuliano Torre, MD (1) · Hanna Mandel, MD (1) · Henkjan J. Verkade, MD, PhD (1) · Ira J Fox, MD (1) · Laurent Servais (1) · Maarten Sinaasappel, MD (1) · Stephanie Grunewald, MD (1) · Suyash Prasad, M.D. (1) · Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM (1)