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 | EUCTR2022-002762-33-DE (EUCTR) | 12/12/2022 | 21/09/2022 | Phase3B/4 extension study to Further Evaluate Safety, Tolerability and Efficacy of AX 250 in Patients with MPS Type IIIB | A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Tralesinidase alfa Other descriptive name: rhNAGLU-IGF2 | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 3;Phase 4 | Turkey;Colombia;Germany;United Kingdom;United States | ||
| 2 | NCT05492799 (ClinicalTrials.gov) | December 2, 2022 | 5/8/2022 | Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE | Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients | MPS III B | Drug: AX 250 | Allievex Corporation | NULL | Enrolling by invitation | N/A | N/A | All | 15 | Phase 4 | United States;Colombia;Germany;Turkey;United Kingdom |
| 3 | EUCTR2017-003083-13-GB (EUCTR) | 02/03/2018 | 11/01/2018 | Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 2 | United States;Taiwan;Spain;Turkey;Germany;Colombia;United Kingdom | ||
| 4 | NCT03784287 (ClinicalTrials.gov) | February 19, 2018 | 30/3/2018 | A Treatment Extension Study of Mucopolysaccharidosis Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB;MPS III B | Drug: AX 250 | Allievex Corporation | NULL | Active, not recruiting | 0 Years | 18 Years | All | 20 | Phase 2 | United States;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom |
| 5 | NCT02754076 (ClinicalTrials.gov) | April 2016 | 27/2/2016 | A Treatment Study of Mucopolysaccharidosis Type IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | MPS III B;Mucopolysaccharidosis Type IIIB | Drug: AX 250 | Allievex Corporation | NULL | Completed | 1 Year | 10 Years | All | 23 | Phase 1/Phase 2 | United States;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom |
| 6 | EUCTR2015-001985-25-DE (EUCTR) | 22/03/2016 | 16/11/2015 | Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Spain;Turkey;Australia;Colombia;Germany;United Kingdom | ||
| 7 | EUCTR2015-001985-25-GB (EUCTR) | 06/11/2015 | 30/09/2015 | Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Spain;Turkey;Australia;Germany;Colombia;United Kingdom | ||
| 8 | EUCTR2017-003083-13-DE (EUCTR) | 24/11/2017 | Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: AX 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | Allievex Corporation | NULL | NA | Female: yes Male: yes | 33 | Phase 2 | United States;Taiwan;Spain;Turkey;Colombia;Germany;United Kingdom |