AT-GTX-501 ( DrugBank: - )

---

1 disease

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	2

19. Lysosomal storage disease

---

Clinical trials : 899 /  Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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