131. Alexander disease
1 clinical trial, 3 drugs (DrugBank: 1 drug), 0 gene, 0 pathway
Searched query = "Alexander disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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-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 |