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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04488133 (ClinicalTrials.gov) | February 26, 2021 | 20/7/2020 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Not yet recruiting | 3 Months | 36 Months | All | 60 | Phase 4 | NULL |
| 2 | EUCTR2019-002663-10-GB (EUCTR) | 17/12/2020 | 01/09/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophy | Escalating 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United 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 | ||
| 3 | NCT04576494 (ClinicalTrials.gov) | December 2020 | 29/9/2020 | Study 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 Evaluation | Spinal Muscular Atrophy | Other: Monthly assessments of functional motor abilities by a trained therapist;Drug: Nusinersen | CHU de Reims | NULL | Not yet recruiting | 18 Years | N/A | All | 24 | N/A | France |
| 4 | NCT04404764 (ClinicalTrials.gov) | May 27, 2020 | 19/5/2020 | Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study | Characterization 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 Atrophy | Drug: Nusinersen Injectable Product | Hospital Israelita Albert Einstein | Ministry of Health, Brazil | Recruiting | 6 Months | N/A | All | 100 | Brazil | |
| 5 | EUCTR2019-002663-10-ES (EUCTR) | 06/05/2020 | 10/05/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United 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. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2019-002663-10-HU (EUCTR) | 23/04/2020 | 05/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 7 | EUCTR2019-002663-10-LV (EUCTR) | 27/03/2020 | 12/02/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 8 | NCT04089566 (ClinicalTrials.gov) | March 26, 2020 | 11/9/2019 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Recruiting | N/A | N/A | All | 152 | Phase 2;Phase 3 | United States;Colombia;Estonia;Hungary;Ireland;Latvia;Spain;Taiwan |
| 9 | NCT04050852 (ClinicalTrials.gov) | July 17, 2019 | 7/8/2019 | Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments | Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments | Spinal Muscular Atrophy | Drug: Nusinersen Treatments | NYU Langone Health | Winthrop University Hospital | Recruiting | 5 Years | 21 Years | All | 10 | Early Phase 1 | United States |
| 10 | NCT03878030 (ClinicalTrials.gov) | April 1, 2019 | 10/3/2019 | Effect of Nusinersen on Adults With Spinal Muscular Atrophy | Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3 | Adult Spinal Muscular Atrophy | Drug: nusinersen | Northwell Health | NULL | Active, not recruiting | 18 Years | 60 Years | All | 12 | United States | |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT04591678 (ClinicalTrials.gov) | October 1, 2018 | 23/10/2019 | Adults With SMA Treated With Nusinersen | Characterizing Longitudinal Outcomes in Adults With SMA Treated With Nusinersen | Spinal Muscular Atrophy | Drug: nusinersen | Ohio State University | Biogen;Cure SMA | Active, not recruiting | 18 Years | 60 Years | All | 15 | United States | |
| 12 | NCT04587492 (ClinicalTrials.gov) | January 1, 2017 | 7/10/2020 | Metabolomics of Children With SMA | Metabolome of Children With Spinal Muscular Atrophy Treated With Nusinersen | Spinal Muscular Atrophy | Drug: Nusinersen | University Medical Centre Ljubljana | NULL | Completed | N/A | 21 Years | All | 35 | Slovenia | |
| 13 | EUCTR2015-001870-16-GB (EUCTR) | 04/10/2016 | 29/10/2015 | A 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 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 14 | EUCTR2015-001870-16-BE (EUCTR) | 29/07/2016 | 23/05/2016 | A 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 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 15 | NCT02865109 (ClinicalTrials.gov) | June 1, 2016 | 10/8/2016 | Expanded 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 Atrophy | Drug: Nusinersen | Biogen | NULL | Available | N/A | N/A | All | China;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. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2015-001870-16-SE (EUCTR) | 26/05/2016 | 22/01/2016 | A 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 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of | ||
| 17 | EUCTR2015-001870-16-DE (EUCTR) | 08/03/2016 | 28/10/2015 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 18 | NCT02594124 (ClinicalTrials.gov) | November 4, 2015 | 30/10/2015 | A 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 396443 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Active, not recruiting | N/A | N/A | All | 292 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan |
| 19 | EUCTR2014-003657-33-DE (EUCTR) | 21/09/2015 | 05/05/2015 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United States;Germany;United Kingdom | ||
| 20 | NCT02462759 (ClinicalTrials.gov) | August 19, 2015 | 14/5/2015 | A 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-CS4 | Spinal Muscular Atrophy | Drug: Nusinersen;Procedure: Sham Procedure | Biogen | NULL | Terminated | N/A | N/A | All | 21 | Phase 2 | United States;Germany |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2014-002098-12-DE (EUCTR) | 05/08/2015 | 09/04/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Canada;Turkey;Australia;Germany;United Kingdom;Italy | ||
| 22 | EUCTR2014-002098-12-GB (EUCTR) | 10/06/2015 | 30/03/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Turkey;Australia;Germany;Italy;United Kingdom | ||
| 23 | NCT02386553 (ClinicalTrials.gov) | May 20, 2015 | 27/2/2015 | A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An 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 Atrophy | Drug: Nusinersen | Biogen | NULL | Active, not recruiting | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom |
| 24 | NCT02292537 (ClinicalTrials.gov) | November 24, 2014 | 12/11/2014 | A 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 Atrophy | Spinal Muscular Atrophy | Drug: Nusinersen;Procedure: Sham procedure | Biogen | NULL | Completed | 2 Years | 12 Years | All | 126 | Phase 3 | United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom |
| 25 | NCT02193074 (ClinicalTrials.gov) | August 19, 2014 | 14/7/2014 | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy | 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 Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen;Procedure: Sham procedure | Biogen | NULL | Terminated | N/A | 210 Days | All | 122 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT02052791 (ClinicalTrials.gov) | January 2014 | 30/1/2014 | An 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-CS10 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | N/A | N/A | All | 47 | Phase 1 | United States |
| 27 | NCT01839656 (ClinicalTrials.gov) | May 8, 2013 | 22/4/2013 | A 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 Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | N/A | 210 Days | All | 21 | Phase 2 | United States;Canada |
| 28 | NCT01780246 (ClinicalTrials.gov) | January 2013 | 28/1/2013 | An 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-CS1 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | 2 Years | 15 Years | All | 18 | Phase 1 | United States |
| 29 | NCT01703988 (ClinicalTrials.gov) | October 2012 | 8/10/2012 | An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | An 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 | Drug: Nusinersen | Biogen | NULL | Completed | 2 Years | 15 Years | All | 34 | Phase 1;Phase 2 | United States |
| 30 | NCT01494701 (ClinicalTrials.gov) | November 30, 2011 | 13/12/2011 | An 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 Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | 2 Years | 14 Years | All | 28 | Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | EUCTR2017-000621-12-Outside-EU/EEA (EUCTR) | 15/02/2017 | A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular Atrophy | 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 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. | NULL | NA | Female: yes Male: yes | 20 | Phase 2 | United States;Canada | |||
| 32 | EUCTR2019-002663-10-GR (EUCTR) | 02/11/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | Canada;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 | |||
| 33 | EUCTR2017-000327-27-Outside-EU/EEA (EUCTR) | 26/01/2017 | An Open-Label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | An 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. | NULL | NA | Female: yes Male: yes | 34 | Phase 2 | United States | |||
| 34 | EUCTR2019-002663-10-PL (EUCTR) | 11/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United 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 | |||
| 35 | EUCTR2019-002663-10-IE (EUCTR) | 04/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United 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 | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2019-002663-10-DE (EUCTR) | 23/10/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating 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 Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | 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;Canada;Argentina;Brazil;Poland;Germany |