LYS
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 104 |
19. ライソゾーム病
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 104 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 | EUCTR2021-005200-35-CZ (EUCTR) | 05/08/202220220805 | 24/05/202220220524 | Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With ... | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL ... | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10 ... | Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR Trade Name: Idursulfase (Elaprase) Product Name: idursulfase (ELAPRASE) INN or Proposed INN: Idursulfase Other descriptive name: Purified form of the lysosomal enzyme iduronate-2-sulfatase Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE ... | Denali Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2;Phase 3 | Colombia;Argentina;United States;Czechia;United Kingdom;Spain;Canada;Czech Republic;Sweden;Netherlands;Turkey;Belgium;Brazil;Mexico;Italy;Australia;France;Germany Colombia;Argentina;United States;Czechia;United Kingdom;Spain;Canada;Czech Republic;Sweden;Netherlan ... | ||
| 2 | EUCTR2019-000667-24-IT (EUCTR) | 28/03/202220220328 | 23/09/202120210923 | A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosid ... | A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease - STAAR A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Toler ... | Fabry Disease (X-linked lysosomal storage disease) MedDRA version: 20.0;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] Fabry Disease (X-linked lysosomal storage disease) MedDRA version: 20.0;Level: PT;Classification cod ... | Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A Product Code: [ST-920] Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA Trade Name: PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE Product Name: Paracetamolo Product Code: [Paracetamolo] INN or Proposed INN: PARACETAMOLO Trade Name: ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE Product Name: difenidramina cloridrato Product Code: [difenidramina cloridrato] INN or Proposed INN: DIFENIDRAMINA CLORIDRATO Trade Name: PREDNISONE DOC GENERICI - 5 MG COMPRESSE 10 COMPRESSE IN BLISTER PVC-PVDC/ALU Product Name: Prednisone Product Code: [Prednisone] INN or Proposed INN: PREDNISONE Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galact ... | Sangamo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 1;Phase 2 | United States;Taiwan;Canada;Australia;Germany;United Kingdom;Italy | ||
| 3 | NCT04283227 (ClinicalTrials.gov) | January 17, 202220220117 | 13/2/202020200213 | OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) | An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD). An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL- ... | Lysosomal Storage Diseases;Metachromatic Leukodystrophy | Genetic: OTL-200 | Orchard Therapeutics | Ospedale San Raffaele | Recruiting | N/A | N/A | All | 6 | Phase 3 | Italy |
| 4 | EUCTR2020-005229-95-NL (EUCTR) | 23/08/202120210823 | 19/03/202120210319 | Study to assess safety, tolerability and efficacy of PBKR03 in pediatric subjects with Krabbe disease (GALax-C) Study to assess safety, tolerability and efficacy of PBKR03 in pediatric subjects with Krabbe diseas ... | A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C) - Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects with Early Infantile Krab A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Toler ... | Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactocerebrosidase; GALC) MedDRA version: 20.0;Level: PT;Classification code 10023492;Term: Krabbe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LS ... | Product Code: PBKR03 INN or Proposed INN: Not available yet Other descriptive name: Adeno-associated virus serotype hu68 containing the human GALC gene Product Code: PBKR03 INN or Proposed INN: Not available yet Other descriptive name: Adeno-associated v ... | Passage Bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 1;Phase 2 | United States;Canada;Brazil;Israel;Netherlands;United Kingdom | ||
| 5 | EUCTR2019-002936-97-FR (EUCTR) | 06/07/202120210706 | 09/02/202120210209 | A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU) A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving th ... | A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU) A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving th ... | MPS IIIB is a devastating lysosomal storage disease, caused by a N-a-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB 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] MPS IIIB is a devastating lysosomal storage disease, caused by a N-a-acetylglucosaminidase (NAGLU) g ... | Product Name: rAAV9.CMV.hNAGLU Product Code: ABO-101 INN or Proposed INN: rAAV9.CMV.hNAGLU Other descriptive name: Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene Product Name: rAAV9.CMV.hNAGLU Product Code: ABO-101 INN or Proposed INN: rAAV9.CMV.hNAGLU Other descri ... | Abeona Therapeutics Europe SL. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | United States;France;Germany | ||
| 6 | NCT04532047 (ClinicalTrials.gov) | July 1, 202120210701 | 19/8/202020200819 | 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 |
| 7 | NCT04273269 (ClinicalTrials.gov) | May 11, 202120210511 | 21/1/202020200121 | A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis | An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human ß-Galactosidase cDNA for Treatment of GM1 Gangliosidosis An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Ca ... | GM1 Gangliosidosis | Genetic: LYS-GM101 | LYSOGENE | NULL | Active, not recruiting | N/A | 3 Years | All | 16 | Phase 1/Phase 2 | United States;France;United Kingdom |
| 8 | NCT04455230 (ClinicalTrials.gov) | September 8, 202020200908 | 29/6/202020200629 | A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 | A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Follo ... | Fabry Disease;Lysosomal Storage Diseases | Genetic: FLT190 | Freeline Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | Male | 50 | Phase 1/Phase 2 | Germany;United Kingdom |
| 9 | EUCTR2019-002979-34-ES (EUCTR) | 18/05/202020200518 | 25/05/202020200525 | 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 th ... | 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 th ... | 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] MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase ge ... | 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 | ||
| 10 | EUCTR2015-003904-21-FR (EUCTR) | 23/12/201920191223 | 11/09/201920190911 | 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] MPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase g ... | Product Name: scAAV9.U1A.hSGSH Product Code: ABO-102 INN or Proposed INN: rebisufligene etisparvovec Other descriptive name: scAAV9.U1A.hSGSH Product Name: scAAV9.U1A.hSGSH Product Code: ABO-102 INN or Proposed INN: rebisufligene etisparvovec Ot ... | Abeona Therapeutics Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;France;Brazil;Spain;Australia;Germany;United Kingdom |