19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
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. | 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 | EUCTR2021-000706-21-DE (EUCTR) | 10/12/2021 | 28/06/2021 | A study to evaluate the long-term efficacy and safety of AT-GTX-501 in patients with CLN6 Batten disease, who have received AT-GTX-501 in a previous study. | LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE | Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease. MedDRA version: 23.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: scAAV9.CB.CLN6 Product Code: AT-GTX-501 INN or Proposed INN: Not yet assigned Other descriptive name: Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 13 | Phase 1;Phase 2 | United States;Germany | ||
| 2 | NCT02725580 (ClinicalTrials.gov) | March 9, 2016 | 16/3/2016 | Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease | Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9 | Variant Late-Infantile Neuronal Ceroid Lipofuscinosis | Genetic: AT-GTX-501 | Amicus Therapeutics | NULL | Completed | 1 Year | N/A | All | 13 | Phase 1/Phase 2 | United States |