3. 脊髄性筋萎縮症
[臨床試験数:179,薬物数:102(DrugBank:26),標的遺伝子数:52,標的パスウェイ数:78

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
7 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-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
2EUCTR2014-003657-33-GB
(EUCTR)
17/11/201519/06/2015A 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]
Biogen Idec Research LimitedNULLNot Recruiting Female: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
3EUCTR2014-003657-33-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
4NCT02462759
(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
5EUCTR2014-002098-12-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2014-002098-12-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
7EUCTR2014-002098-12-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