19. Lysosomal storage disease Clinical trials / Disease details
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 |