Synthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
2筋萎縮性側索硬化症2
127前頭側頭葉変性症2

2. 筋萎縮性側索硬化症


臨床試験数 : 645 薬物数 : 589 - (DrugBank : 163) / 標的遺伝子数 : 150 - 標的パスウェイ数 : 225
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-005193-94-BE
(EUCTR)
26/01/202215/10/2021Phase 1b/2a Study of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1;Classification code 10068968;Term: Frontotemporal dementia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: WVE-004
INN or Proposed INN: not yet assigned
Other descriptive name: Synthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts
Wave Life Sciences UK LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2United States;Canada;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom;New Zealand;Sweden
2EUCTR2020-005193-94-SE
(EUCTR)
26/01/202212/10/2021Phase 1b/2a Study of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1;Classification code 10068968;Term: Frontotemporal dementia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: WVE-004
INN or Proposed INN: not yet assigned
Other descriptive name: Synthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts
Wave Life Sciences UK LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2United States;Canada;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom;New Zealand;Sweden

127. 前頭側頭葉変性症


臨床試験数 : 90 薬物数 : 87 - (DrugBank : 30) / 標的遺伝子数 : 39 - 標的パスウェイ数 : 88
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-005193-94-SE
(EUCTR)
26/01/202212/10/2021Phase 1b/2a Study of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1;Classification code 10068968;Term: Frontotemporal dementia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: WVE-004
INN or Proposed INN: not yet assigned
Other descriptive name: Synthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts
Wave Life Sciences UK LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2United States;Canada;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom;New Zealand;Sweden
2EUCTR2020-005193-94-BE
(EUCTR)
26/01/202215/10/2021Phase 1b/2a Study of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1;Classification code 10068968;Term: Frontotemporal dementia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: WVE-004
INN or Proposed INN: not yet assigned
Other descriptive name: Synthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts
Wave Life Sciences UK LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2United States;Canada;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom;New Zealand;Sweden