3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 237 / Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 75
Showing 1 to 10 of 23 diseases
| 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 | 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 A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinerse ... | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | 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 United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Ja ... | ||
| 2 | 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 A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinerse ... | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | 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 United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Ja ... | ||
| 3 | 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 A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinerse ... | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | Biogen Idec Research Limited | NULL | Not Recruiting | 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 United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Ja ... | ||
| 4 | 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 extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 3 ... | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | 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 United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Ja ... | ||
| 5 | EUCTR2015-001870-16-ES (EUCTR) | 08/03/2016 | 03/02/2016 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 3 ... | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product ... | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 274 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Ger ... | |||
| 6 | EUCTR2013-004422-29-BE (EUCTR) | 22/10/2015 | 04/08/2015 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with i ... | 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 A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spin ... | 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 Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product ... | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Ja ... | ||
| 7 | 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 Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular at ... | 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 A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolera ... | 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] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | 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) Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | Biogen Idec Research Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United States;Germany;United Kingdom | ||
| 8 | 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 A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Pre ... | 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 An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple D ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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) Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oli ... | 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 | ||
| 9 | EUCTR2014-001947-18-DE (EUCTR) | 07/07/2015 | 02/12/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with l ... | 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 A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spin ... | 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 Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product ... | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sw ... | ||
| 10 | EUCTR2014-002098-12-IT (EUCTR) | 12/05/2015 | 24/03/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Pre ... | 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 An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple D ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spin ... | 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) Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product ... | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | United States;Qatar;Taiwan;Argentina;Turkey;Australia;Israel;Germany;United Kingdom;Italy |