Ambroxol ( DrugBank: Ambroxol )

2 diseases

ID	Disease name (Link within this page)	Number of trials
6	Parkinson disease	4
19	Lysosomal storage disease	8

6. Parkinson disease

Clinical trials : 2,307 / Drugs : 2,007 - (DrugBank : 349) / Drug target genes : 188 - Drug target pathways : 199

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	NCT05287503 (ClinicalTrials.gov)	February 15, 2022	28/1/2022	Ambroxol as a Disease-modifying Treatment in GBA-PD	Ambroxol as a Disease-modifying Treatment to Reduce the Risk of Cognitive Impairment in GBA-associated Parkinson's Disease. A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 2 Trial	Parkinson Disease;GBA Gene Mutation	Drug: Ambroxol Hydrochloride;Drug: Placebo	Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta	University of Campania Luigi Vanvitelli;IRCCS National Neurological Institute C. Mondino Foundation	Recruiting	21 Years	80 Years	All	60	Phase 2	Italy
2	NCT02941822 (ClinicalTrials.gov)	December 2016	5/9/2016	Ambroxol in Disease Modification in Parkinson Disease	A Phase IIA Prospective, Single-Centre, Open Label Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of Ambroxol in Patients With Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease	Parkinson Disease	Drug: Ambroxol	University College, London	The Cure Parkinson's Trust	Completed	40 Years	80 Years	All	23	Phase 2	United Kingdom
3	EUCTR2015-002571-24-GB (EUCTR)	14/10/2016	12/02/2019	A Pilot Study to Evaluate the Safety, Tolerability (any side effects of the drug) and Pharmacodynamic (biochemical and physiological effects of drugs). The effects of Ambroxol in Patients with Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease.	A Phase IIA Prospective, Single-Centre, Open Label Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of Ambroxol in Patients with Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease - AiM-PD	Parkinson disease;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: AMBROSAN 60mg Tablets Product Name: Ambroxol INN or Proposed INN: Ambroxol hydrochloride	Joint Research Office	NULL	Not Recruiting			Female: yes Male: yes	20	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no	United Kingdom
4	NCT02914366 (ClinicalTrials.gov)	November 2015	8/9/2016	Ambroxol as a Treatment for Parkinson's Disease Dementia	Ambroxol as a Novel Disease Modifying Treatment for Parkinson's Disease Dementia	Parkinson's Disease Dementia	Drug: Ambroxol;Other: Placebo	Lawson Health Research Institute	Weston Brain Institute;Western University, Canada;London Health Sciences Centre	Active, not recruiting	50 Years	N/A	All	55	Phase 2	Canada

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	EUCTR2021-002550-82-NL (EUCTR)	22/12/2022	30/07/2022	Effectiveness of ambroxol for treating children and adults with Gaucher disease type 3	Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3	Gaucher disease type 3 MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metabolic and nutritional disorders congenital;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL Hydrochloride Other descriptive name: Mucoangin Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL HYDROCHLORIDE Other descriptive name: AMBROXOL HYDROCHLORIDE PH. EUR.	Amsterdam UMC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	4	Phase 3	Netherlands
2	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.	Yutaka Shunichiro	NULL	Complete	>= 20age old	< 35age old	Male	16	Phase 1	Japan
3	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
4	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
5	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	Completed	18 Years	75 Years	All	40	Phase 2	Israel
6	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
7	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	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	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
8	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	Complete	Not applicable	Not applicable	Both	25	Phase 2	Japan