OAV101 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy12

3. Spinal muscular atrophy


Clinical trials : 237 Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 75
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05386680
(ClinicalTrials.gov)
January 12, 202318/5/2022Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or RisdiplamPhase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to 12 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)Spinal Muscular AtrophyGenetic: OAV101Novartis PharmaceuticalsNULLRecruiting2 Years12 YearsAll28Phase 3Canada;Japan;Netherlands;Spain
2EUCTR2021-003474-31-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
3NCT05335876
(ClinicalTrials.gov)
December 19, 202212/4/2022Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsLong-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical TrialsSpinal Muscular Atrophy (SMA)Biological: onasemnogene abeparvovecNovartis PharmaceuticalsNULLRecruitingN/AN/AAll260Phase 3Australia;Belgium
4NCT05089656
(ClinicalTrials.gov)
January 12, 202211/10/2021Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are = 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never AmbulatoryType 2 Spinal Muscular AtrophyGenetic: OAV101;Procedure: Sham controlNovartis PharmaceuticalsNULLRecruiting2 Years17 YearsAll125Phase 3United States;China;Denmark;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;Vietnam
5NCT05073133
(ClinicalTrials.gov)
November 4, 202120/9/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)A Phase IV 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 From Latin America and Canada With Spinal Muscular Atrophy (SMA) - OFELIAMuscular Atrophy, SpinalGenetic: OAV101Novartis PharmaceuticalsNULLActive, not recruitingN/A24 MonthsAll16Phase 4Argentina;Brazil
6EUCTR2020-005995-37-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
7EUCTR2020-005995-37-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
8NCT04851873
(ClinicalTrials.gov)
September 8, 20216/4/2021Safety 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)Spinal Muscular AtrophyGenetic: OAV101Novartis PharmaceuticalsNULLActive, not recruitingN/A17 YearsAll24Phase 3United States;Australia;Belgium;Canada;France;Italy;Portugal;Taiwan;United Kingdom;Switzerland
9EUCTR2020-005995-37-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
10EUCTR2020-005995-37-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
11EUCTR2021-003474-31-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
12EUCTR2021-006781-21-DK
(EUCTR)
20/12/2022Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical TrialsLong-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials 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 vg (vector genomes)/mL solution for infusion
Product Name: OAV101
Product Code: OAV101
Trade Name: Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
Product Name: OAV101
Product Code: OAV101
Novartis Pharma AGNULLNAFemale: yes
Male: yes
260Phase 4United States;Saudi Arabia;Taiwan;Greece;Thailand;Spain;Colombia;Switzerland;Italy;India;France;Malaysia;Viet Nam;Australia;Denmark;South Africa;Netherlands;China;United Kingdom;Egypt;Mexico;Canada;Belgium;Brazil;Singapore;Germany;Japan