131. アレキサンダー病 Alexander disease Clinical trials / Disease details


臨床試験数 : 4 薬物数 : 4 - (DrugBank : 1) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-000976-40-IT
(EUCTR)
07/10/202102/08/2021Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients withAlexander DiseaseA Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics ofIntrathecally Administered ION373 in Patients with Alexander Disease - NA Alexander 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: ION373
Product Code: [ION373]
INN or Proposed INN: ION373
Other descriptive name: 2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING
IONIS PHARMACEUTICALS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2;Phase 3United States;Canada;Argentina;Australia;Israel;Netherlands;United Kingdom;Japan;Italy
2NCT04849741
(ClinicalTrials.gov)
June 1, 202116/4/2021A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients With Alexander DiseaseAlexander DiseaseDrug: ION373;Drug: PlaceboIonis Pharmaceuticals, Inc.NULLRecruiting2 Years65 YearsAll58Phase 3United States;Canada;Netherlands
3EUCTR2020-000976-40-NL
(EUCTR)
04/05/202112/02/2021Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander DiseaseA Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease Alexander Disease
MedDRA version: 22.1;Level: PT;Classification code 10083059;Term: Alexander disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ION373
Product Code: ION373
INN or Proposed INN: ION373
Other descriptive name: 2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING GLIAL FIBRILLARY ACIDIC PROTEIN MESSENGER RIBONUCLEIC ACID
Ionis PharmaceuticalsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1;Phase 2;Phase 3United States;Canada;Argentina;Australia;Israel;Netherlands;United Kingdom;Italy;Japan
4EUCTR2020-000976-40-GB
(EUCTR)
28/07/2020Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander DiseaseA Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease Alexander Disease
MedDRA version: 22.1;Level: PT;Classification code 10083059;Term: Alexander disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ION373
Product Code: ION373
INN or Proposed INN: ION373
Other descriptive name: 2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING GLIAL FIBRILLARY ACIDIC PROTEIN MESSENGER RIBONUCLEIC ACID
Ionis Pharmaceuticals, IncNULLNAFemale: yes
Male: yes
42Phase 1;Phase 2;Phase 3United States;Canada;Argentina;Australia;Israel;Netherlands;Japan;Italy;United Kingdom