19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
2 trials found
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agemin
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PhaseCountries
1NCT01801709
(ClinicalTrials.gov)
June 201428/1/2013Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic LeukodystrophyA Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.Metachromatic LeukodystrophyGenetic: intracerebral administration of AAVrh.10cuARSAInstitut National de la Santé Et de la Recherche Médicale, FranceEuropean Leukodystrophy Association;Assistance Publique - Hôpitaux de ParisActive, not recruiting6 Months5 YearsAll5Phase 1/Phase 2France
2EUCTR2011-004410-42-FR
(EUCTR)
26/11/201219/12/2012Intracerebral Gene Therapy for MLDA phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA cDNA to children with metachromatic leukodystrophy - Intracerebral Gene Therapy for MLD Early onset forms of MLD
MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 14.1;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AAVrh.10cuARSA
Product Code: Non applicable
InsermNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 1/2France