131. Alexander disease Clinical trials / Disease details
Clinical trials : 4 / Drugs : 4 - (DrugBank : 1) / Drug target gene : 0 - Drug target pathway : 0
| 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 | EUCTR2020-000976-40-IT (EUCTR) | 07/10/2021 | 02/08/2021 | Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients withAlexander Disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 1;Phase 2;Phase 3 | United States;Canada;Argentina;Australia;Israel;Netherlands;United Kingdom;Japan;Italy | ||
| 2 | NCT04849741 (ClinicalTrials.gov) | June 1, 2021 | 16/4/2021 | A 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 Disease | Alexander Disease | Drug: ION373;Drug: Placebo | Ionis Pharmaceuticals, Inc. | NULL | Recruiting | 2 Years | 65 Years | All | 73 | Phase 3 | United States;Australia;Canada;Israel;Italy;Japan;Netherlands;United Kingdom |
| 3 | EUCTR2020-000976-40-NL (EUCTR) | 04/05/2021 | 12/02/2021 | Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease | 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 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 | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 1;Phase 2;Phase 3 | United States;Canada;Israel;Netherlands;United Kingdom;Italy;Japan | ||
| 4 | EUCTR2020-000976-40-GB (EUCTR) | 28/07/2020 | Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease | 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 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, Inc | NULL | NA | Female: yes Male: yes | 42 | Phase 1;Phase 2;Phase 3 | United States;Canada;Argentina;Australia;Israel;Netherlands;Japan;Italy;United Kingdom |