Branaplam ( DrugBank: Branaplam )


2 diseases
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy3
8Huntington disease7

3. Spinal muscular atrophy


Clinical trials : 237 Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 75
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02268552
(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
2EUCTR2014-002053-19-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 AGNULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
3EUCTR2014-002053-19-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 AGNULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy

8. Huntington disease


Clinical trials : 242 Drugs : 205 - (DrugBank : 62) / Drug target genes : 85 - Drug target pathways : 159
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-000105-92-LT
(EUCTR)
02/03/202228/10/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: LMI070
INN or Proposed INN: BRANAPLAM
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2France;United States;Hungary;Canada;Spain;Belgium;Lithuania;Russian Federation;Germany;United Kingdom;Italy
2NCT05111249
(ClinicalTrials.gov)
December 8, 202115/10/2021A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study With Open-Label Extension to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of LMI070/Branaplam Administered as Weekly Oral Doses in Participants With Early Manifest Huntington's DiseaseEarly Manifest Huntington DiseaseDrug: Branaplam;Drug: PlaceboNovartis PharmaceuticalsNULLActive, not recruiting25 Years75 YearsAll75Phase 2United States;Belgium;Canada;France;Germany;Hungary;Italy;Spain;United Kingdom
3EUCTR2020-000105-92-IT
(EUCTR)
26/11/202120/09/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington's Disease - - Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: [LMI070]
INN or Proposed INN: BRANAPLAM
Product Name: branaplam
Product Code: [LMI070]
INN or Proposed INN: BRANAPLAM
Product Name: branaplam
Product Code: [LMI070]
INN or Proposed INN: BRANAPLAM
Product Name: branaplam
Product Code: [LMI070]
INN or Proposed INN: BRANAPLAM
Product Name: branaplam
Product Code: [LMI070]
INN or Proposed INN: BRANAPLAM
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2France;United States;Hungary;Canada;Spain;Belgium;Lithuania;Russian Federation;Germany;United Kingdom;Italy
4EUCTR2020-000105-92-FR
(EUCTR)
18/11/202103/09/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: LMI070
INN or Proposed INN: BRANAPLAM
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2Spain;Belgium;Lithuania;Russian Federation;Germany;United Kingdom;Italy;United States;France;Hungary;Canada
5EUCTR2020-000105-92-ES
(EUCTR)
02/11/202126/08/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: LMI070
INN or Proposed INN: BRANAPLAM
Novartis Farmacéutica S.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2France;United States;Hungary;Canada;Belgium;Spain;Lithuania;Russian Federation;Germany;United Kingdom;Italy
6EUCTR2020-000105-92-HU
(EUCTR)
29/10/202124/08/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: LMI070
INN or Proposed INN: BRANAPLAM
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2France;United States;Hungary;Canada;Belgium;Spain;Lithuania;Russian Federation;Germany;United Kingdom;Italy
7EUCTR2020-000105-92-DE
(EUCTR)
15/10/202118/08/2021A study to evaluate the safety, tolerability and efficacy of weekly oral doses of branaplam in patients with Huntington's DiseaseA Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease Huntington's disease
MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: branaplam
Product Code: LMI070
INN or Proposed INN: BRANAPLAM
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 2United States;France;Hungary;Canada;Spain;Belgium;Lithuania;Russian Federation;Germany;United Kingdom;Italy