3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
| 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 | EUCTR2014-002053-19-BG (EUCTR) | 04/10/2018 | 31/07/2018 | Clinical 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 AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy | ||
| 2 | EUCTR2014-002053-19-HU (EUCTR) | 17/09/2018 | 17/07/2018 | Clinical 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 Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy | ||
| 3 | EUCTR2014-002053-19-CZ (EUCTR) | 14/02/2018 | 30/10/2017 | Clinical 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 AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 4 | NCT02268552 (ClinicalTrials.gov) | April 2, 2015 | 1/10/2014 | An 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 Atrophy | Spinal Muscular Atrophy | Drug: branaplam | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 182 Days | All | 40 | Phase 1/Phase 2 | Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands |
| 5 | EUCTR2014-002053-19-IT (EUCTR) | 01/04/2015 | 27/10/2014 | Clinical 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] | Product Name: NA Product Code: LMI070 INN or Proposed INN: NA Other descriptive name: NA | NOVARTIS FARMA S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy | ||
| 6 | EUCTR2014-002053-19-DE (EUCTR) | 02/02/2015 | 30/10/2014 | Clinical 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 AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy | ||
| 7 | EUCTR2014-002053-19-DK (EUCTR) | 16/12/2014 | 15/10/2014 | Clinical 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 AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 8 | EUCTR2014-002053-19-BE (EUCTR) | 15/12/2014 | 04/11/2014 | Clinical 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 AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 9 | EUCTR2014-002053-19-PL (EUCTR) | 03/01/2018 | Clinical 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 AG | NULL | NA | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | |||
| 10 | EUCTR2014-002053-19-NL (EUCTR) | 27/11/2014 | Clinical 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;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: LMI070 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Belgium;Denmark;Germany;Netherlands;Italy |