AVXS-101 (previously known as scAAV9.CB.SMN) ( DrugBank: - )


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14

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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-000266-29-NL
(EUCTR)
25/03/201905/06/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot Recruiting Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden
2EUCTR2017-004087-35-NL
(EUCTR)
28/02/201909/07/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
44Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Netherlands;Germany;Japan
3EUCTR2017-004087-35-BE
(EUCTR)
31/01/201916/05/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
29Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan
4EUCTR2017-000266-29-BE
(EUCTR)
31/01/201923/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;United Kingdom;Italy;Sweden
5EUCTR2017-004087-35-IT
(EUCTR)
24/12/201823/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
44Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan
6EUCTR2017-004087-35-ES
(EUCTR)
21/12/201820/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
44 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan
7EUCTR2017-000266-29-ES
(EUCTR)
18/12/201828/05/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy;Sweden
8EUCTR2017-000266-29-DE
(EUCTR)
05/11/201805/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3France;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Sweden
9EUCTR2017-004087-35-GB
(EUCTR)
24/08/201808/05/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
Novartis Gene Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
29Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan
10EUCTR2017-000266-29-GB
(EUCTR)
12/06/201812/02/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
33Phase 3France;Spain;Belgium;Netherlands;Italy;United Kingdom;Sweden
11EUCTR2017-000266-29-IT
(EUCTR)
21/05/201809/06/2020Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - N/A Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
AVEXIS, INC.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
12EUCTR2017-000266-29-FR
(EUCTR)
05/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
13EUCTR2017-000266-29-SE
(EUCTR)
12/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
14EUCTR2017-004087-35-DE
(EUCTR)
06/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNAFemale: yes
Male: yes
27Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan