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1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 163 |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Showing 1 to 10 of 163 trials
| 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) | 22/12/202220221222 | 30/07/202220220730 | 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] Gaucher disease type 3 MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metabolic ... | 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. Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL Hydrochloride Other descriptive nam ... | Amsterdam UMC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Phase 3 | Netherlands | ||
| 2 | JPRN-jRCT2071220022 | 14/07/202220220714 | 25/06/202220220625 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry's disease | Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Agalsidase alfa Pharmaceutical form: Concentrate for solution for infusion, Route of administration: IV infusion Drug: Agalsidase beta (GZ419828) Pharmaceutical form: Powder for concentrate for solution for infusion, Route of administration: IV infusion Drug: Migalastat Pharmaceutical form: Hard capsules, Route of administration: Oral Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Agalsidas ... | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | <= 65age old | Both | 90 | Phase 3 | China;Italy;Republic of Korea;Japan |
| 3 | EUCTR2021-002350-90-AT (EUCTR) | 22/04/202220220422 | 28/10/202120211028 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and f ... | A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of ven ... | MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | Sanofi-Aventis Recherche & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;A ... | ||
| 4 | EUCTR2021-005356-10-NL (EUCTR) | 08/04/202220220408 | 18/01/202220220118 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo ... | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ... | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other des ... | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Spain;Australia;Germany;Netherlands;United Kingdom;Switzerland | ||
| 5 | EUCTR2021-002350-90-FI (EUCTR) | 01/04/202220220401 | 06/10/202120211006 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and f ... | A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months - PERIDOT A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of ven ... | MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | Sanofi-Aventis Recherche & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;A ... | ||
| 6 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/202220220323 | 05/01/202220220105 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher ... | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator stu ... | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher ... | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Veng ... | Sanofi-aventis recherche & développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2 | Phase 3 | Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentin ... | ||
| 7 | EUCTR2021-005356-10-SK (EUCTR) | 15/03/202220220315 | 28/12/202120211228 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo ... | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ... | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other des ... | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerland United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerla ... | ||
| 8 | EUCTR2021-002350-90-PL (EUCTR) | 08/03/202220220308 | 28/10/202120211028 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and f ... | A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months - PERIDOT A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of ven ... | MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | Sanofi-Aventis Recherche & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | France;Mexico;Canada;Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China France;Mexico;Canada;Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;A ... | ||
| 9 | EUCTR2021-005356-10-DE (EUCTR) | 04/03/202220220304 | 29/12/202120211229 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo ... | Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neona ... | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other des ... | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Czech Republic;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Switzerland United States;Czechia;Czech Republic;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Swi ... | ||
| 10 | NCT05228145 (ClinicalTrials.gov) | January 31, 202220220131 | 17/12/202120211217 | Gene Therapy Study for Children With CLN5 Batten Disease | A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease A Phase 1/2 Intracerebroventricular and Intravitreal Administrationof NGN-101 for Treatment of Neuro ... | Neuronal Ceroid Lipofuscinosis CLN5 | Genetic: NGN-101 | Neurogene Inc. | NULL | Recruiting | 3 Years | 9 Years | All | 6 | Phase 1/Phase 2 | United States;United Kingdom |