Nusinersen    (DrugBank: Nusinersen)

1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy36

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.
36 / 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
2EUCTR2019-002663-10-GB
(EUCTR)
17/12/202001/09/2020Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nursinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: Nursinersen
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
3NCT04576494
(ClinicalTrials.gov)
December 202029/9/2020Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms): a Multicenter Single-case Experimental Design in Multiple Baselines Across Subjects, Randomized, Single-blinded EvaluationSpinal Muscular AtrophyOther: Monthly assessments of functional motor abilities by a trained therapist;Drug: NusinersenCHU de ReimsNULLNot yet recruiting18 YearsN/AAll24N/AFrance
4NCT04404764
(ClinicalTrials.gov)
May 27, 202019/5/2020Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational StudyCharacterization of the Clinical-epidemiological Profile of Patients With Spinal Muscular Atrophy (SMA) 5q Types II and III in Follow-up in the Brazilian Unified Public Health System: A Cross-sectional Observational Study (Registry)Spinal Muscular AtrophyDrug: Nusinersen Injectable ProductHospital Israelita Albert EinsteinMinistry of Health, BrazilRecruiting6 MonthsN/AAll100Brazil
5EUCTR2019-002663-10-ES
(EUCTR)
06/05/202010/05/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-002663-10-HU
(EUCTR)
23/04/202005/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
7EUCTR2019-002663-10-LV
(EUCTR)
27/03/202012/02/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
8NCT04089566
(ClinicalTrials.gov)
March 26, 202011/9/2019Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: NusinersenBiogenNULLRecruitingN/AN/AAll152Phase 2;Phase 3United States;Colombia;Estonia;Hungary;Ireland;Latvia;Spain;Taiwan
9NCT04050852
(ClinicalTrials.gov)
July 17, 20197/8/2019Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen TreatmentsPulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen TreatmentsSpinal Muscular AtrophyDrug: Nusinersen TreatmentsNYU Langone HealthWinthrop University HospitalRecruiting5 Years21 YearsAll10Early Phase 1United States
10NCT03878030
(ClinicalTrials.gov)
April 1, 201910/3/2019Effect of Nusinersen on Adults With Spinal Muscular AtrophyEffect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3Adult Spinal Muscular AtrophyDrug: nusinersenNorthwell HealthNULLActive, not recruiting18 Years60 YearsAll12United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT04591678
(ClinicalTrials.gov)
October 1, 201823/10/2019Adults With SMA Treated With NusinersenCharacterizing Longitudinal Outcomes in Adults With SMA Treated With NusinersenSpinal Muscular AtrophyDrug: nusinersenOhio State UniversityBiogen;Cure SMAActive, not recruiting18 Years60 YearsAll15United States
12NCT04587492
(ClinicalTrials.gov)
January 1, 20177/10/2020Metabolomics of Children With SMAMetabolome of Children With Spinal Muscular Atrophy Treated With NusinersenSpinal Muscular AtrophyDrug: NusinersenUniversity Medical Centre LjubljanaNULLCompletedN/A21 YearsAll35Slovenia
13EUCTR2015-001870-16-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
14EUCTR2015-001870-16-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
15NCT02865109
(ClinicalTrials.gov)
June 1, 201610/8/2016Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)Infantile-onset Spinal Muscular AtrophyDrug: NusinersenBiogenNULLAvailableN/AN/AAllChina;Colombia;Mexico;New Zealand;Taiwan;Turkey;Australia;Austria;Belgium;Canada;Denmark;Finland;France;Germany;Greece;Ireland;Israel;Italy;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Slovenia;Spain;Sweden;Switzerland;United Kingdom;United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2015-001870-16-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
17EUCTR2015-001870-16-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
18NCT02594124
(ClinicalTrials.gov)
November 4, 201530/10/2015A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443Spinal Muscular AtrophyDrug: nusinersenBiogenNULLActive, not recruitingN/AN/AAll292Phase 3United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan
19EUCTR2014-003657-33-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
20NCT02462759
(ClinicalTrials.gov)
August 19, 201514/5/2015A Study to Assess the Safety and Tolerability of Nusinersen (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-CS4Spinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham ProcedureBiogenNULLTerminatedN/AN/AAll21Phase 2United States;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2014-002098-12-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
22EUCTR2014-002098-12-GB
(EUCTR)
10/06/201530/03/2015A 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: 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: Spinraza
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;Turkey;Australia;Germany;Italy;United Kingdom
23NCT02386553
(ClinicalTrials.gov)
May 20, 201527/2/2015A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically 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.Spinal Muscular AtrophyDrug: NusinersenBiogenNULLActive, not recruitingN/A6 WeeksAll25Phase 2United States;Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom
24NCT02292537
(ClinicalTrials.gov)
November 24, 201412/11/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)A 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 AtrophySpinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham procedureBiogenNULLCompleted2 Years12 YearsAll126Phase 3United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom
25NCT02193074
(ClinicalTrials.gov)
August 19, 201414/7/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With 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 AtrophySpinal Muscular AtrophyDrug: nusinersen;Procedure: Sham procedureBiogenNULLTerminatedN/A210 DaysAll122Phase 3United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02052791
(ClinicalTrials.gov)
January 201430/1/2014An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/AN/AAll47Phase 1United States
27NCT01839656
(ClinicalTrials.gov)
May 8, 201322/4/2013A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/A210 DaysAll21Phase 2United States;Canada
28NCT01780246
(ClinicalTrials.gov)
January 201328/1/2013An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years15 YearsAll18Phase 1United States
29NCT01703988
(ClinicalTrials.gov)
October 20128/10/2012An 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 AtrophySpinal Muscular AtrophyDrug: NusinersenBiogenNULLCompleted2 Years15 YearsAll34Phase 1;Phase 2United States
30NCT01494701
(ClinicalTrials.gov)
November 30, 201113/12/2011An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years14 YearsAll28Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2019-002663-10-PL
(EUCTR)
11/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
32EUCTR2019-002663-10-GR
(EUCTR)
02/11/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3Canada;Argentina;Brazil;Poland;Germany;United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico
33EUCTR2017-000621-12-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
34EUCTR2019-002663-10-IE
(EUCTR)
04/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
35EUCTR2019-002663-10-DE
(EUCTR)
23/10/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
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
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2017-000327-27-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