Otl-200 dispersion for infusion (DrugBank: -)
1 disease告示番号 | 疾患名(ページ内リンク) | 臨床試験数 |
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19 | ライソゾーム病 | 1 |
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | EUCTR2017-001730-26-IT (EUCTR) | 30/11/2017 | 08/04/2019 | Gene therapy study using a frozen formulation of OTL-200 in patients with Metachromatic Leukodystrophy (MLD). | A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic Leukodystrophy (MLD). - A clinical study using cryopreserved OTL-200 for treatment of MLD. | Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: OTL-200 Dispersion for Infusion Product Code: [OTL-200] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 3 | Italy |