19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Showing 1 to 10 of 132 diseases
| 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 | NCT05222906 (ClinicalTrials.gov) | April 18, 2022 | 20/1/2022 | 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) A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Stu ... | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 40 | Phase 3 | United States;Argentina;Canada;China;France;Germany;Hungary;Japan |
| 2 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/2022 | 05/01/2022 | 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 ... | ||
| 3 | NCT05152914 (ClinicalTrials.gov) | November 1, 2021 | 29/11/2021 | Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 | Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Intravitreal Enzyme Replacement Therapyto Prevent Retinal Disease Progression in Children With Neuro ... | Neuronal Ceroid Lipofuscinosis Type 2 | Drug: Cerliponase Alfa | David L Rogers, MD | NULL | Enrolling by invitation | 24 Months | 72 Months | All | 5 | Phase 1/Phase 2 | United States |
| 4 | NCT05134571 (ClinicalTrials.gov) | October 28, 2021 | 21/10/2021 | China Post-marketing Surveillance (PMS) Study of Aldurazyme® | A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Re ... | Mucopolysaccharidosis I | Drug: Laronidase | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 5 Years | N/A | All | 12 | Phase 4 | China |
| 5 | NCT05054387 (ClinicalTrials.gov) | October 13, 2021 | 9/9/2021 | China Post-marketing Surveillance (PMS) Study of Fabrazyme® | A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replaceme ... | Fabry's Disease | Drug: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 8 Years | N/A | All | 22 | Phase 4 | China |
| 6 | JPRN-jRCT2031210279 | 15/09/2021 | 27/08/2021 | An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme re ... | An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme re ... | Acid sphingomyelinase deficiency | Drug: Olipudase alfa (GZ402665) Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion. Drug: Olipudase alfa(GZ402665) Pharmaceutical form: lyophilized powder, Route of administration: Intr ... | Tanaka Tomoyuki | NULL | Not Recruiting | >= 20age old | Not applicable | Both | 1 | Phase 2-3 | Japan |
| 7 | NCT04877132 (ClinicalTrials.gov) | July 21, 2021 | 3/5/2021 | Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapyfor Patients With Chronic Aci ... | Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapyfor Patients With Chronic Aci ... | Sphingomyelin Lipidosis | Drug: olipudase alfa (GZ402665) | Sanofi | NULL | Approved for marketing | 3 Years | N/A | All | NULL | ||
| 8 | NCT04532047 (ClinicalTrials.gov) | July 1, 2021 | 19/8/2020 | In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases | In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). In Utero Enzyme Replacement Therapy(ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs) ... | MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease Infantile-Onset;Wolman Disease MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease In ... | Drug: Aldurazyme (laronidase) | University of California, San Francisco | Duke University | Recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 9 | NCT04281537 (ClinicalTrials.gov) | March 1, 2020 | 7/2/2020 | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry D ... | A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase Beta A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their C ... | Fabry Disease | Drug: Agalsidase Beta;Drug: Agalsidase Alpha | Amicus Therapeutics | NULL | Completed | 18 Years | N/A | All | 76 | United States;Brazil;Japan;Taiwan;Turkey | |
| 10 | JPRN-jRCT2051190085 | 06/01/2020 | 19/12/2019 | A long-term study of beta-glucuronidase enzyme replacement therapy for Sly disease | A long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease A long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enz ... | Mucopolysaccharidosis type VII Mucopolysaccharidosis VII, Sly syndrome, beta-glucuronidase, vestronidase alfa;D016538 Mucopolysaccharidosis type VII Mucopolysaccharidosis VII, Sly syndrome, beta-glucuronidase, vestroni ... | Investigational drug: Vestronidase alfa [Recombinant human beta-glucuronidase (rhGUS)], 4 mg/kg, IV, QOW Investigational drug: Vestronidase alfa[Recombinant human beta-glucuronidase(rhGUS)], 4 mg/kg, IV, Q ... | Hamazaki Takashi | NULL | Recruiting | Not applicable | Not applicable | Both | 4 | Phase 3 | Japan |