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 | NCT04360265 (ClinicalTrials.gov) | September 28, 2020 | 20/4/2020 | A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102 | A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH) | Mucopolysaccharidosis III-A | Biological: ABO-102 | Ultragenyx Pharmaceutical Inc | Abeona Therapeutics, Inc | Enrolling by invitation | N/A | N/A | All | 50 | United States;Australia;Spain | |
| 2 | EUCTR2019-002979-34-ES (EUCTR) | 18/05/2020 | 25/05/2020 | A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH) | A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH) | MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. 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: scAAV9.U1A.hSGSH Product Code: ABO-102 INN or Proposed INN: Rebisufligene etisparvovec | Abeona Therapeutics Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 34 | Phase 1;Phase 2 | Germany;United States;France;Spain;Australia | ||
| 3 | EUCTR2015-003904-21-FR (EUCTR) | 23/12/2019 | 11/09/2019 | Gene transfer clinical trial for Mucopolysaccharidosis IIIA | Phase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA | MPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment currently available for the disease. 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: scAAV9.U1A.hSGSH Product Code: ABO-102 INN or Proposed INN: rebisufligene etisparvovec Other descriptive name: scAAV9.U1A.hSGSH | Abeona Therapeutics Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Brazil;Spain;Australia;Germany;United Kingdom | ||
| 4 | NCT04088734 (ClinicalTrials.gov) | September 18, 2019 | 11/9/2019 | Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease | MPS IIIA;Sanfilippo Syndrome;Sanfilippo A;Mucopolysaccharidosis III | Drug: ABO-102 | Ultragenyx Pharmaceutical Inc | Abeona Therapeutics, Inc | Terminated | 2 Years | 18 Years | All | 5 | Phase 1/Phase 2 | United States;Australia;Spain |
| 5 | EUCTR2018-000504-42-ES (EUCTR) | 21/05/2019 | 09/04/2018 | Gene Therapy Clinical Trial for Mucopolysaccharidosis IIIA in patient with middle and advanced phases of the disease | A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease - ABO-102 for Treatment of Middle and Advanced Phases of MPS IIIA | MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, withdeterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. 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: scAAV9.U1A.SGSH Product Code: ABO-102 INN or Proposed INN: scAAV9.U1A.SGSH Other descriptive name: scAAV9.U1A.SGSH | Abeona Therapeutics Inc | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;Spain;Australia | ||
| 6 | NCT02716246 (ClinicalTrials.gov) | March 2016 | 17/3/2016 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA | MPS IIIA;Sanfilippo Syndrome;Sanfilippo A;Mucopolysaccharidosis III | Biological: ABO-102 | Ultragenyx Pharmaceutical Inc | Abeona Therapeutics, Inc | Active, not recruiting | N/A | N/A | All | 28 | Phase 1/Phase 2 | United States;Australia;Spain;France;Germany |