Onasemnogene Abeparvovec-xioi ( DrugBank: Onasemnogene abeparvovec, onasemnogene abeparvovec-xioi )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
3	脊髄性筋萎縮症	5

3. 脊髄性筋萎縮症

臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

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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	NCT04042025 (ClinicalTrials.gov)	February 10, 2020	31/7/2019	Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi	A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving ...	Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMA	Biological: Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	NULL	Active, not recruiting	N/A	N/A	All	85	Phase 3	United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;S ...
2	NCT03837184 (ClinicalTrials.gov)	May 31, 2019	8/2/2019	Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One ...	Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients Wi ...	Spinal Muscular Atrophy Type I	Biological: Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	PRA Health Sciences	Completed	0 Days	6 Months	All	2	Phase 3	Japan;Korea, Republic of;Taiwan
3	NCT03505099 (ClinicalTrials.gov)	April 10, 2018	13/4/2018	Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioiin Spinal Muscular Atrophy (SMA) f ...	A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnose ...	Spinal Muscular Atrophy	Biological: onasemnogene abeparvovec-xioi	Novartis Gene Therapies	PRA Health Sciences	Completed	N/A	42 Days	All	30	Phase 3	United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;Un ...
4	NCT03381729 (ClinicalTrials.gov)	December 14, 2017	13/12/2017	Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy	Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With ...	Spinal Muscular Atrophy	Biological: Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	NULL	Completed	6 Months	60 Months	All	32	Phase 1	United States
5	NCT03421977 (ClinicalTrials.gov)	September 21, 2017	30/1/2018	Long-Term Follow-up Study for Patients From AVXS-101-CL-101	A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clini ...	Spinal Muscular Atrophy 1	Biological: Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	NULL	Active, not recruiting	N/A	N/A	All	13		United States

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