Previously termed sc.AAV9.CB.SMN and AVXS-101 ( 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6

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
1EUCTR2021-003474-31-GR
(EUCTR)
03/01/202315/09/2022Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma
INN or Proposed INN: Onasemnogene abeparvovec
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Trade Name: Okrido 6mg/mL oral solution
Product Name: Okrido
INN or Proposed INN: PREDNISOLONE SODIUM PHOSPHATE
Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
125Phase 3United States;United Arab Emirates;Saudi Arabia;Taiwan;Greece;Thailand;Russian Federation;Colombia;Italy;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Viet Nam;Denmark;South Africa;China
2EUCTR2020-005995-37-PT
(EUCTR)
15/10/202119/07/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 3United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
3EUCTR2020-005995-37-IT
(EUCTR)
28/09/202117/08/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Prednisolone
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Trade Name: Prednisolone
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Trade Name: Zolgensma
Product Name: OAV101
Product Code: [AVXS-101]
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: Previously termed sc.AAV9.CB.SMN and AVXS-101
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
4EUCTR2020-005995-37-BE
(EUCTR)
03/09/202128/06/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 3Portugal;France;United States;Taiwan;Canada;Belgium;Australia;Germany;United Kingdom;Italy;Switzerland
5EUCTR2020-005995-37-FR
(EUCTR)
25/08/202121/06/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
6EUCTR2021-003474-31-DK
(EUCTR)
02/11/2021Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER Spinal Muscular Atrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma
Product Name: OAV101
Product Code: OAV101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Trade Name: Okrido 6mg/mL oral solution
Product Name: Okrido
INN or Proposed INN: prednisolone sodium phosphate
Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE
Novartis Pharma AGNULLNAFemale: yes
Male: yes
125Phase 3United States;United Arab Emirates;Saudi Arabia;Taiwan;Thailand;Russian Federation;Colombia;Italy;Vietnam;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Denmark;South Africa;China