| ROUBERTIE, Agathe |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
NCT04003922: Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term |
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| Recruiting | N/A | 40 | Europe | Adalimumab treatment | Assistance Publique Hopitaux De Marseille | Rasmussen Encephalitis | 04/24 | 10/25 | | |
| Recruiting | N/A | 200 | Europe | Survey | Assistance Publique Hopitaux De Marseille | Epileptic Encephalopathy | 09/25 | 09/25 | | |
DOPADEF, NCT05211609: Prevalence of High Plasmatic 3OMethyldopa Level in a Specific Population of Patients With a Symptomatology Compatible With AADC Deficiency |
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| Recruiting | N/A | 388 | Europe | Plasmatic 3-O-Methyldopa dosage | University Hospital, Montpellier | Aromatic L-Amino Acid Decarboxylase Deficiency | 05/24 | 11/24 | | |
| Recruiting | N/A | 1000 | Europe | | Institut National de la Santé Et de la Recherche Médicale, France | Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Mucopolysaccharidosis IX, Multiple Sulfatase Deficiency Disease | 12/26 | 12/26 | | |
| Guffon, Nathalie |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
NCT06031259: Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment |
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| Recruiting | 2/3 | 8 | Europe, Canada | Idursulfase-IT, Elaprase | Takeda | Hunter Syndrome | 01/34 | 06/34 | | |
| Recruiting | N/A | 30 | Europe, US | | Phoenix Nest, Vaincres Les Maladies Lysosomales, Association Sanfilippo Sud, Aparito Ltd. | Sanfilippo Syndrome Type C | 07/27 | 11/27 | | |
| Lampe, Christina |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) |
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| Recruiting | 2/3 | 54 | Europe, Canada, US, RoW | tividenofusp alfa, idursulfase | Denali Therapeutics Inc. | Mucopolysaccharidosis II | 12/25 | 12/25 | | |
| Balwierz, Walentyna |
ChIm-NB-PL, NCT05272371 / 2021-003832-96: Immunotherapy With Dinutuximab Beta in Combination With Chemotherapy for the Treatment of Patients With Primary Neuroblastoma Refractory to Standard Therapy and With Relapsed or Progressive Disease |
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| Recruiting | 1 | 20 | Europe | Chemoimmunotherapy (Dinutuximab beta in combination with chemotherapy) | Jagiellonian University, Medicinal Research Agency | High-Risk Neuroblastoma | 09/26 | 12/26 | | |
NCT04253015: A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta |
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| Recruiting | N/A | 125 | Europe | Data-collection | EusaPharma (UK) Limited, United BioSource, LLC | Neuroblastoma | 03/32 | 06/32 | | |
| Deubzer, Heidi |
NCT04253015: A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta |
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| Recruiting | N/A | 125 | Europe | Data-collection | EusaPharma (UK) Limited, United BioSource, LLC | Neuroblastoma | 03/32 | 06/32 | | |
| Wieczorek, Aleksandra |
NCT04253015: A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta |
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| Recruiting | N/A | 125 | Europe | Data-collection | EusaPharma (UK) Limited, United BioSource, LLC | Neuroblastoma | 03/32 | 06/32 | | |
| Wieczore, Aleksandra |
NCT04253015: A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta |
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| Recruiting | N/A | 125 | Europe | Data-collection | EusaPharma (UK) Limited, United BioSource, LLC | Neuroblastoma | 03/32 | 06/32 | | |
| Muschol, Nichole |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
| Recruiting | 1/2 | 12 | Europe | JR-441 | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis III-A | 10/29 | 10/29 | | |
| Zebrowska, Urszula |
NCT04253015: A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta |
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| Recruiting | N/A | 125 | Europe | Data-collection | EusaPharma (UK) Limited, United BioSource, LLC | Neuroblastoma | 03/32 | 06/32 | | |
| Bik-Multanowski, Miroslaw |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
