OTL-200

( DrugBank: - / KEGG DRUG: - )

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	5

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

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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
1	NCT04283227 (ClinicalTrials.gov)	January 17, 2022	13/2/2020	OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)	An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL- ...	Lysosomal Storage Diseases;Metachromatic Leukodystrophy	Genetic: OTL-200	Orchard Therapeutics	Ospedale San Raffaele	Recruiting	N/A	N/A	All	6	Phase 3	Italy
2	EUCTR2019-002636-82-IT (EUCTR)	02/12/2019	30/07/2019	A clinical trial to evaluate the effect of OTL-200, a gene therapy treatment in patients with Late J ...	An open label, non-randomised trial to evaluate the safety and efficacy of a single infusion of OTL- ...	Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metac ...	Product Code: OTL-200 INN or Proposed INN: Not Applicable Other descriptive name: OTL-200Dispersion fo ...	Orchard Therapeutics (Europe) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	6	Phase 3	Italy
3	NCT03392987 (ClinicalTrials.gov)	January 25, 2018	12/12/2017	A Safety and Efficacy Study of Cryopreserved OTL-200for Treatment of Metachromatic Leukodystrophy (M ...	A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Len ...	Lysosomal Storage Disease;Metachromatic Leukodystrophy	Genetic: OTL-200	Orchard Therapeutics	Ospedale San Raffaele	Active, not recruiting	N/A	6 Years	All	10	Phase 2	Italy
4	EUCTR2017-001730-26-IT (EUCTR)	30/11/2017	08/04/2019	Gene therapy study using a frozen formulation of OTL-200in patients with Metachromatic Leukodystroph ...	A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with len ...	Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metac ...	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
5	NCT01560182 (ClinicalTrials.gov)	April 9, 2010	16/3/2012	Gene Therapy for Metachromatic Leukodystrophy (MLD)	A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromat ...	Lysosomal Storage Disease;Metachromatic Leukodystrophy	Genetic: OTL-200 Gene Therapy	Orchard Therapeutics	Ospedale San Raffaele	Active, not recruiting	N/A	7 Years	All	20	Phase 1/Phase 2	Italy

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