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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
10 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2014-002053-19-BG
(EUCTR)
04/10/201831/07/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy
2EUCTR2014-002053-19-HU
(EUCTR)
17/09/201817/07/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44Phase 1;Phase 2United States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy
3EUCTR2014-002053-19-CZ
(EUCTR)
14/02/201830/10/2017Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
4NCT02268552
(ClinicalTrials.gov)
April 2, 20151/10/2014An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular AtrophySpinal Muscular AtrophyDrug: branaplamNovartis PharmaceuticalsNULLActive, not recruitingN/A182 DaysAll40Phase 1;Phase 2Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands
5EUCTR2014-002053-19-IT
(EUCTR)
01/04/201527/10/2014Clinical trial of LMI070 given my mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]NOVARTIS FARMA S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
22Phase 1;Phase 2United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2014-002053-19-DE
(EUCTR)
02/02/201530/10/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44Phase 1;Phase 2Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy
7EUCTR2014-002053-19-DK
(EUCTR)
16/12/201415/10/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Not established
Other descriptive name: LMI070
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
8EUCTR2014-002053-19-BE
(EUCTR)
15/12/201404/11/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Branaplam
Other descriptive name: LMI070
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
9EUCTR2014-002053-19-PL
(EUCTR)
03/01/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Branaplam
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNAFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
10EUCTR2014-002053-19-NL
(EUCTR)
27/11/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patientsAn open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Code: LMI070
INN or Proposed INN: LMI070
Novartis Pharma Services AGNULLNAFemale: yes
Male: yes
22Phase 1;Phase 2United States;Belgium;Denmark;Germany;Netherlands;Italy