19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

7 trials found

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	JPRN-jRCT2051210024	10/06/2021	11/05/2021	Investigation of the Food Effect on the Pharmacokinetics of JT408T	Investigation of the Food Effect on the Pharmacokinetics of JT408T	Improvement of neurological symptoms of Gaucher's disease	1) After fasting, administer 400 mg of ambroxol hydrochloride orally in a single dose. 2) After meals, administer 400 mg of ambroxol hydrochloride orally in a single dose.	Ioroi Minoru	NULL	Not Recruiting	>= 20age old	< 35age old	Male	16	Phase 1	Japan
2	JPRN-jRCTs061190017	21/11/2019	26/09/2019	Japan-Ambroxol Chaperone Study	2 cohort, Non-randomized, Multi-site Study to Evaluate the Efficacy and Safety of Chaperone Therapy with Ambroxol Hydrochloride in Patients with Neuronopathic Gaucher disease - J-ACT	Neuronopathic Gaucher disease	Ambroxol will be given.	Narita Aya	NULL	Recruiting	Not applicable	Not applicable	Both	25	Phase 3	Japan
3	JPRN-JMA-IIA00421	07/05/2019	26/04/2019	Phase II/III Study to Evaluate the Efficacy and Safety of Chaperone Therapy with Ambroxol Hydrochloride (JT408T) in Patients with Neuronopathic Gaucher disease (Japan- Lysosomal Optimization Study: J-LO study)	Phase II/III Study to Evaluate the Efficacy and Safety of Chaperone Therapy with Ambroxol Hydrochloride (JT408T) in Patients with Neuronopathic Gaucher disease (Japan- Lysosomal Optimization Study: J-LO study)	Neuronopathic Gaucher disease	Intervention type:DRUG. Intervention1:ADMINISTRATION, Dose form:TABLET, Route of administration:ORAL.	Tottori University Hospital, Departrment of Child Neurology	NULL	Completed	No Limit	No Limit	BOTH	3	Phase 2-3	Japan
4	NCT03950050 (ClinicalTrials.gov)	March 1, 2019	6/8/2018	Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy	Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy	Gaucher Disease, Type 1	Drug: Ambroxol	Shaare Zedek Medical Center	NULL	Recruiting	18 Years	75 Years	All	60	Phase 2	Israel
5	NCT01463215 (ClinicalTrials.gov)	December 2012	27/10/2011	Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease	An Open-Label, Dose Escalation With 2 Dose Levels, Proof-of-Concept Clinical Trial of Ambroxol for the Treatment of Type I Gaucher Disease	Type I Gaucher Disease	Drug: Ambroxol	Exsar Corporation	NULL	Suspended	16 Years	N/A	Both	20	Phase 1/Phase 2	United States
6	JPRN-UMIN000009392	2012/11/24	24/11/2012	An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol		Neuronopathic Gaucher disease	Ambroxol at a dose level of 25 mg/kg/day (upper limit: 1g/day) will be given by mouth for 6 months.	Tottori University, Faculty of Medicine, Institute of Neurological Science	NULL	Recruiting	Not applicable	Not applicable	Male and Female	5	Phase 2,3	Japan
7	JPRN-jRCTs061180090	16/03/2010	22/03/2019	Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol	An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol - Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol	Neuronopathic Gaucher disease Gaucher disease, Pharmacological chaperone therapy, ambroxol;D005776	Ambroxol at a dose level of 25 mg/kg/day (upper limit: 1300mg/day) will be given by mouth for 6 months.	Maegaki Yoshihiro	NULL	Not Recruiting	Not applicable	Not applicable	Both	25	Phase 2	Japan