AT-GTX-501 ( DrugBank: - )

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
19ライソゾーム病2

19. ライソゾーム病

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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2021-000706-21-DE
(EUCTR)		10/12/202128/06/2021A 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.NULLNot RecruitingFemale: yes
Male: yes		13Phase 1;Phase 2United States;Germany
2NCT02725580
(ClinicalTrials.gov)		March 9, 201616/3/2016Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) DiseasePhase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9Variant Late-Infantile Neuronal Ceroid LipofuscinosisGenetic: AT-GTX-501Amicus TherapeuticsNULLCompleted1 YearN/AAll13Phase 1/Phase 2United States