Intracerebral administration of AAVrh.10cuARSA ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1

19. ライソゾーム病


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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
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