Survival of motor neuron 2 (smn2) splicing modulator antisense oligonucleotide    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy23

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.
23 / 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
1EUCTR2015-001870-16-GB
(EUCTR)
04/10/201629/10/2015A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
2EUCTR2015-001870-16-BE
(EUCTR)
29/07/201623/05/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
3EUCTR2015-001870-16-SE
(EUCTR)
26/05/201622/01/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
4EUCTR2015-001870-16-ES
(EUCTR)
08/03/201603/02/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
274United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
5EUCTR2015-001870-16-DE
(EUCTR)
08/03/201628/10/2015An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2013-004422-29-BE
(EUCTR)
22/10/201504/08/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
7EUCTR2014-003657-33-DE
(EUCTR)
21/09/201505/05/2015A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA).A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE 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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLNot RecruitingFemale: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
8EUCTR2014-002098-12-DE
(EUCTR)
05/08/201509/04/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
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] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Qatar;Taiwan;Canada;Turkey;Australia;Germany;United Kingdom;Italy
9EUCTR2014-001947-18-DE
(EUCTR)
07/07/201502/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
10EUCTR2014-002098-12-IT
(EUCTR)
12/05/201524/03/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25United States;Qatar;Taiwan;Argentina;Turkey;Australia;Israel;Germany;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2014-001947-18-ES
(EUCTR)
08/04/201509/02/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
12EUCTR2014-001947-18-IT
(EUCTR)
12/03/201512/05/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;United Kingdom;Italy;Korea, Republic of;Sweden
13EUCTR2014-001947-18-SE
(EUCTR)
03/02/201523/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
14EUCTR2013-004422-29-ES
(EUCTR)
20/01/201508/09/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3Korea, Republic of;United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden
15EUCTR2013-004422-29-DE
(EUCTR)
08/01/201527/11/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2013-004422-29-SE
(EUCTR)
09/12/201423/10/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
17EUCTR2013-004422-29-GB
(EUCTR)
01/12/201429/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy - Study to assess the Clinical Efficacy and Safety of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
18EUCTR2013-004422-29-IT
(EUCTR)
13/11/201423/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
19EUCTR2017-000327-27-Outside-EU/EEA
(EUCTR)
26/01/2017An Open-Label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyAn Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: nusinersen
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
34Phase 2United States
20EUCTR2017-000621-12-Outside-EU/EEA
(EUCTR)
15/02/2017A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular AtrophyA Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: NUSINERSEN
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
20Phase 2United States;Canada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2015-001870-16-FR
(EUCTR)
03/05/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443. Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
274Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
22EUCTR2014-001947-18-GB
(EUCTR)
19/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
23EUCTR2014-001947-18-FR
(EUCTR)
18/06/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
117Phase 3United States;France;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden