IB1001 ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病13
65原発性免疫不全症候群3

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-005356-10-NL
(EUCTR)
08/04/202218/01/2022A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type CEffects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-acetyl-L-leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 3United States;Czechia;Slovakia;Spain;Australia;Germany;Netherlands;United Kingdom;Switzerland
2EUCTR2021-005356-10-SK
(EUCTR)
15/03/202228/12/2021A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type CEffects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-acetyl-L-leucine
Product Code: IB1001
INN or Proposed INN: N Acetyl L leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 3United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerland
3EUCTR2021-005356-10-DE
(EUCTR)
04/03/202229/12/2021A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type CEffects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Product Name: N-acetyl-L-leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 3United States;Czechia;Czech Republic;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Switzerland
4NCT03759639
(ClinicalTrials.gov)
September 4, 201927/11/2018N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.Niemann-Pick Disease, Type CDrug: IB1001IntraBio IncNULLActive, not recruiting6 YearsN/AAll34Phase 2United States;Germany;Slovakia;Spain;United Kingdom
5EUCTR2018-004331-71-GB
(EUCTR)
20/06/201914/01/2019To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with Niemann-Pick Type C disease (NPC).Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational,multicenter, open-label, rater-blinded Phase II study. - IB1001-201Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients withNiemann-Pick Type C disease (NPC).
MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: SUB195712
Other descriptive name: N-Acetyl-L-Leucine
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: N-Acetyl-L-Leucine
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;United Kingdom;Slovakia;Germany;Spain
6EUCTR2018-004406-25-GB
(EUCTR)
20/06/201906/02/2020To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and SandhoffDisease): A multinational, multicenter, open-label, rater-blinded Phase II study - IB1001-202 To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).
MedDRA version: 20.0;Level: PT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 21.1;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: SUB195712
Other descriptive name: N-Acetyl-L-Leucine
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: N-Acetyl-L-Leucine
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Spain;Germany;United Kingdom
7EUCTR2018-004331-71-ES
(EUCTR)
10/06/201922/01/2019A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease.Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N Acetyl L Leucine
Product Code: IB1001
INN or Proposed INN: N Acetyl L leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
39 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Slovakia;Spain;Germany;United Kingdom
8EUCTR2018-004406-25-ES
(EUCTR)
10/06/201912/04/2019A safety and efficacy study of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study. GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N Acetyl L leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
39 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Spain;Germany;United Kingdom
9NCT03759665
(ClinicalTrials.gov)
June 7, 201927/11/2018N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)Effects of N-Acetyl-L-Leucine on GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II StudyGM2 Gangliosidosis;Tay-Sachs Disease;Sandhoff DiseaseDrug: IB1001IntraBio IncNULLCompleted6 YearsN/AAll30Phase 2United States;Germany;Spain;United Kingdom
10EUCTR2018-004331-71-SK
(EUCTR)
04/06/201930/01/2019A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease.Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational,multicenter, open-label, rater-blinded Phase II study. Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Slovakia;Spain;Germany;United Kingdom
11EUCTR2018-004406-25-DE
(EUCTR)
09/05/201923/01/2019A safety and efficacy study of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study. GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Spain;Germany;United Kingdom
12EUCTR2018-004331-71-DE
(EUCTR)
08/05/201902/01/2019A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease.Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Slovakia;Spain;Germany;United Kingdom
13EUCTR2021-005356-10-CZ
(EUCTR)
15/07/2022A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type CEffects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Niemann-Pick Disease type C (NPC)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-acetyl-L-leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L Leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLNAFemale: yes
Male: yes
53Phase 3United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerland

65. 原発性免疫不全症候群


臨床試験数 : 500 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004407-39-DE
(EUCTR)
26/06/202005/02/2020A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia.Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study. Ataxia-Telangiectasia (A-T)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 2United States;Spain;Germany;United Kingdom
2NCT03759678
(ClinicalTrials.gov)
January 8, 202027/11/2018N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II StudyAtaxia Telangiectasia;Louis Bar SyndromeDrug: IB1001IntraBio IncNULLRecruiting6 YearsN/AAll39Phase 2United States;Germany;Spain;United Kingdom
3EUCTR2018-004407-39-GB
(EUCTR)
18/09/201906/08/2019A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-TelangiectasiaEffects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study. - IB1001-203 To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning, and quality of life in patients with Ataxia-Telangiectasia (A-T);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Product Name: N-Acetyl-L-Leucine
Product Code: IB 1001
Other descriptive name: N-Acetyl-L-Leucine
IntraBio Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
39 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Germany;United Kingdom