Onasemnogene abeparvovec    (DrugBank: Onasemnogene abeparvovec)

1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy17

3. Spinal muscular atrophy    [ 179 clinical trials,   102 drugs,   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways]
Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
17 / 179 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04488133
(ClinicalTrials.gov)
February 26, 202120/7/2020A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecMuscular Atrophy, SpinalDrug: NusinersenBiogenNULLNot yet recruiting3 Months36 MonthsAll60Phase 4NULL
2EUCTR2019-002611-26-FR
(EUCTR)
16/09/202010/03/2020not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
308Phase 3United States;France;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of
3EUCTR2019-002611-26-GB
(EUCTR)
30/03/202031/12/2019not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
308Phase 3United States;France;Taiwan;Canada;Spain;Belgium;Australia;Japan;Italy;United Kingdom;Korea, Republic of
4NCT04042025
(ClinicalTrials.gov)
February 10, 202031/7/2019Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiA Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMABiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLEnrolling by invitationN/AN/AAll308Phase 4United States;Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;Korea, Republic of
5NCT03837184
(ClinicalTrials.gov)
May 15, 20198/2/2019Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesPhase 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 InfusionSpinal Muscular Atrophy Type IBiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsPRA Health SciencesActive, not recruitingN/A6 MonthsAll2Phase 3Japan;Korea, Republic of;Taiwan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2017-000266-29-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
7EUCTR2017-004087-35-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
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
9NCT03461289
(ClinicalTrials.gov)
August 29, 20184/3/2018Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1European, Phase 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 InfusionSMABiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.NULLCompletedN/A6 MonthsAll33Phase 3Belgium;France;Italy;United Kingdom;Germany;Netherlands;Spain;Sweden
10EUCTR2017-004087-35-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2017-000266-29-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
12NCT03505099
(ClinicalTrials.gov)
April 10, 201813/4/2018Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2A 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 SMN2Spinal Muscular AtrophyBiological: onasemnogene abeparvovec-xioiNovartis PharmaceuticalsPRA Health SciencesActive, not recruitingN/A42 DaysAll30Phase 3United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom
13NCT03381729
(ClinicalTrials.gov)
December 14, 201713/12/2017Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyPhase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular AtrophySpinal Muscular AtrophyBiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLSuspended6 Months60 MonthsAll51Phase 1United States
14NCT03306277
(ClinicalTrials.gov)
October 24, 20172/10/2017Gene Replacement Therapy Clinical Trial for Participants 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 InfusionSMA - Spinal Muscular Atrophy;Gene TherapyBiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.NULLCompletedN/A180 DaysAll22Phase 3United States
15NCT03421977
(ClinicalTrials.gov)
September 21, 201730/1/2018Long-Term Follow-up Study for Patients From AVXS-101-CL-101A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101Spinal Muscular Atrophy 1Biological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLActive, not recruitingN/AN/AAll13United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2017-004087-35-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
17EUCTR2017-000266-29-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