19. Lysosomal storage disease
784 clinical trials, 673 drugs (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT02376751 (ClinicalTrials.gov) | August 2015 | 25/2/2015 | An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency | AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY | Lysosomal Acid Lipase Deficiency | Drug: sebelipase alfa | Alexion Pharmaceuticals | NULL | No longer available | 8 Months | N/A | Both | N/A | NULL | |
| 2 | EUCTR2014-000533-22-FI (EUCTR) | 23/06/2015 | 05/06/2015 | Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD). | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;Finland;Italy;United Kingdom | ||
| 3 | EUCTR2011-004287-30-NL (EUCTR) | 29/05/2015 | 01/04/2015 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Netherlands;Germany | ||
| 4 | EUCTR2011-002750-31-CZ (EUCTR) | 16/02/2015 | 22/03/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Germany;Japan;Sweden | |||
| 5 | EUCTR2011-004287-30-GB (EUCTR) | 19/01/2015 | 11/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 18.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Poland;Brazil;Croatia;Australia;Denmark;Netherlands;Germany;Japan | |||
| 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 |
| 6 | EUCTR2014-000533-22-IT (EUCTR) | 18/09/2014 | 22/07/2014 | Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD). | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 17.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;Finland;United Kingdom;Italy | ||
| 7 | EUCTR2011-004287-30-IT (EUCTR) | 18/09/2014 | 18/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 16.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Poland;Croatia;Australia;Denmark;Netherlands;Germany;Japan | ||
| 8 | EUCTR2011-004287-30-DE (EUCTR) | 12/08/2014 | 13/05/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuitcals Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Germany;Netherlands;Japan | ||
| 9 | EUCTR2011-004287-30-BE (EUCTR) | 22/07/2014 | 26/05/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 17.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Netherlands;Germany;Japan | ||
| 10 | EUCTR2011-004287-30-HR (EUCTR) | 07/07/2014 | 03/09/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Alexion Pharmaceuitcals Inc | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Netherlands;Germany;Japan | |||
| 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 |
| 11 | NCT02112994 (ClinicalTrials.gov) | June 24, 2014 | 20/3/2014 | Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency | A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency | Drug: Sebelipase Alfa | Alexion Pharmaceuticals | NULL | Completed | 8 Months | N/A | All | 31 | Phase 2 | United States;Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom |
| 12 | EUCTR2011-004287-30-DK (EUCTR) | 19/06/2014 | 03/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals inc | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Poland;Brazil;Croatia;Denmark;Australia;Netherlands;Germany;Japan | ||
| 13 | NCT02193867 (ClinicalTrials.gov) | June 6, 2014 | 7/7/2014 | Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency | Drug: Sebelipase Alfa | Alexion Pharmaceuticals | NULL | Terminated | N/A | 8 Months | All | 10 | Phase 2 | United States;Finland;Italy;United Kingdom |
| 14 | EUCTR2011-004287-30-ES (EUCTR) | 28/05/2014 | 04/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 16.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipasa Alfa Product Code: SBC-102 INN or Proposed INN: sebelipasa alfa Other descriptive name: lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA); USAN: sebelipasa alfa | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Netherlands;Germany;Japan;United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Poland;Croatia;Australia;Denmark | ||
| 15 | EUCTR2014-000533-22-GB (EUCTR) | 28/05/2014 | 24/04/2014 | Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD). | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 18.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;Finland;Italy;United Kingdom | |||
| 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 |
| 16 | EUCTR2011-002750-31-HR (EUCTR) | 17/07/2013 | 21/05/2014 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 17.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Israel;Chile;Russian Federation;Italy;France;Cyprus;Denmark;Australia;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Sweden | ||
| 17 | EUCTR2011-000032-28-IE (EUCTR) | 12/07/2013 | 16/05/2013 | A research study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats | An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase D | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 19.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 MedDRA version: 19.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 19.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102, recombinant human lysosomal acid lipase INN or Proposed INN: sebelipase alfa Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Alexion Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2;Phase 3 | France;Egypt;United States;Saudi Arabia;Taiwan;Ireland;Turkey;Germany;Italy;United Kingdom | ||
| 18 | EUCTR2011-002750-31-GR (EUCTR) | 26/06/2013 | 15/05/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 16.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Germany;Sweden | |||
| 19 | EUCTR2011-000032-28-DE (EUCTR) | 31/05/2013 | 28/06/2011 | A research study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats | An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 15.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 MedDRA version: 15.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 15.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Sebelipase alfa Product Code: SBC-102, recombinant human lysosomal acid lipase INN or Proposed INN: sebelipase alfa Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | United States;France;Taiwan;Germany;United Kingdom;Italy | |||
| 20 | EUCTR2011-002750-31-DE (EUCTR) | 29/05/2013 | 29/01/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004915 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Germany;Japan;Sweden | |||
| 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 |
| 21 | EUCTR2011-002750-31-ES (EUCTR) | 06/05/2013 | 06/03/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 15.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Belgium;Poland;Brazil;Romania;Croatia;Germany;Sweden | ||
| 22 | EUCTR2011-002750-31-IT (EUCTR) | 15/04/2013 | 25/02/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 14.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Germany;Sweden | |||
| 23 | EUCTR2011-002750-31-GB (EUCTR) | 18/03/2013 | 11/02/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 19.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Chile;Israel;Russian Federation;Italy;France;Cyprus;Denmark;Australia;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Japan;Sweden | ||
| 24 | NCT01757184 (ClinicalTrials.gov) | January 22, 2013 | 17/12/2012 | Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency | A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency | Drug: Sebelipase Alfa;Drug: Placebo | Alexion Pharmaceuticals | NULL | Completed | 4 Years | N/A | All | 66 | Phase 3 | United States;Argentina;Australia;Croatia;Czechia;France;Germany;Italy;Japan;Mexico;Poland;Russian Federation;Spain;Turkey;United Kingdom;Brazil;Canada;Czech Republic;Greece;Israel |
| 25 | EUCTR2011-000032-28-IT (EUCTR) | 07/08/2012 | 03/09/2012 | A reasarch study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that break down fats | AN OPEN LABEL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SBC-102 IN CHILDREN WITH GROWTH FAILURE DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY - SBC-102 in Children with Growth Failure due to LYSOSOMAL ACID LIPASE DEFICIENCY | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 15.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 15.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 15.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | SYNAGEVA BIOPHARMA CORP. | NULL | Not Recruiting | Female: yes Male: yes | 10 | United States;Taiwan;Ireland;Germany;United Kingdom;Italy | |||
| 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 |
| 26 | NCT01488097 (ClinicalTrials.gov) | December 12, 2011 | 26/11/2011 | Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency | An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01 | Cholesterol Ester Storage Disease (CESD);Lysosomal Acid Lipase Deficiency;LAL-Deficiency | Drug: sebelipase alfa | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 8 | Phase 2 | United States;Canada;Czechia;France;United Kingdom;Czech Republic |
| 27 | EUCTR2011-002880-42-GB (EUCTR) | 05/12/2011 | 12/09/2011 | A research study to look at the safety, effectiveness and the long-term effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats who were previously treated with SBC-102 | An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102 - Extension to LAL-CL03 in children with LAL deficiency | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 14.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 10 | United States;France;Germany;United Kingdom | |||
| 28 | EUCTR2011-001513-13-CZ (EUCTR) | 10/11/2011 | 12/09/2011 | A research study to look at the long-term safety, effectiveness and the effects on the body of a new drug, SBC-102, in adults with liver problems caused by a deficiency in the enzyme that breaks down fats. This is an extension study for those subjects who previously received treatment in study LAL-CL01. | AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WHO PREVIOUSLY RECEIVED TREATMENT IN STUDY LAL-CL01 - SBC-102 in patients with liver dysfunction due to LAL deficiency | Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cell types. MedDRA version: 14.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | United States;France;Czech Republic;United Kingdom | ||
| 29 | NCT01473875 (ClinicalTrials.gov) | November 2011 | 10/11/2011 | Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 | An Open Label Multicenter Extension Study to Evaluate the Long-term Efficacy and Safety of SBC-102 in Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 | Lysosomal Acid Lipase Deficiency;Wolman Disease | Drug: SBC-102 | Alexion Pharmaceuticals | NULL | Terminated | N/A | N/A | All | 10 | Phase 2;Phase 3 | France;United Kingdom |
| 30 | EUCTR2011-001513-13-GB (EUCTR) | 24/10/2011 | 31/08/2011 | A research study to look at the long-term safety, effectiveness and the effects on the body of a new drug, SBC-102, in adults with liver problems caused by a deficiency in the enzyme that breaks down fats. This is an extension study for those subjects who previously received treatment in study LAL-CL01. | AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WHO PREVIOUSLY RECEIVED TREATMENT IN STUDY LAL-CL01 - Extension to LAL-CL01 in patients with liver dysfunction due to LAL deficiency | Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cell types. MedDRA version: 14.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | France;United States;Czech Republic;United Kingdom | |||
| 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 |
| 31 | EUCTR2010-024068-16-CZ (EUCTR) | 26/07/2011 | 26/07/2011 | A research study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in adults with liver problems caused by a deficiency in the enzyme that breaks down fats. | An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency. - SBC-102 in patients with liver dysfunction due to LAL deficiency | Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cell types. MedDRA version: 14.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 9 | United Kingdom;Czech Republic;United States | |||
| 32 | NCT01371825 (ClinicalTrials.gov) | May 4, 2011 | 9/6/2011 | Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency | An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency | Lysosomal Acid Lipase Deficiency;Wolman Disease | Drug: Sebelipase alfa (SBC-102) | Alexion Pharmaceuticals | NULL | Completed | N/A | 24 Months | All | 9 | Phase 2;Phase 3 | United States;Egypt;France;Ireland;United Kingdom;Germany;India;Italy;Saudi Arabia;Taiwan;Turkey |
| 33 | NCT01307098 (ClinicalTrials.gov) | April 25, 2011 | 1/3/2011 | Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency | An Open-Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Participants With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD);Lysosomal Acid Lipase Deficiency;LAL-Deficiency | Drug: Sebelipase alfa 0.35 mg/kg;Drug: Sebelipase alfa 1 mg/kg;Drug: Sebelipase alfa 3 mg/kg | Alexion Pharmaceuticals | NULL | Completed | 18 Years | 65 Years | All | 9 | Phase 1;Phase 2 | United States;Czechia;France;United Kingdom;Czech Republic |
| 34 | EUCTR2011-000032-28-GB (EUCTR) | 18/04/2011 | 17/01/2011 | A research study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats | An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 14.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 1;Phase 2;Phase 3 | France;United States;Taiwan;Ireland;Germany;Italy;United Kingdom | ||
| 35 | EUCTR2010-024068-16-GB (EUCTR) | 08/03/2011 | 10/01/2011 | SBC-102 in patients with liver dysfunction due to LAL deficiency | An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency. - SBC-102 in patients with liver dysfunction due to LAL deficiency | Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cell types. MedDRA version: 14.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 9 | Czech Republic;United Kingdom | |||
| 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 |
| 36 | EUCTR2011-000032-28-FR (EUCTR) | 11/04/2011 | A research study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats | An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 13.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 13.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 1;Phase 2 | United States;France;Ireland;Germany;Italy;United Kingdom | |||
| 37 | EUCTR2011-002880-42-FR (EUCTR) | 19/09/2011 | A research study to look at the safety, effectiveness and the long-term effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats who were previously treated with SBC-102 | An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102 - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency | Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). MedDRA version: 14.0;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 14.0;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: recombinant human lysosomal acid lipase (rhLAL) Product Code: SBC-102 INN or Proposed INN: not available Other descriptive name: recombinant human lysosomal acid lipase (rhLAL) | Synageva Biopharma Corp. | NULL | NA | Female: yes Male: yes | 6 | Phase 2 | United States;France;Germany;United Kingdom | |||
| 38 | EUCTR2011-002750-31-FR (EUCTR) | 12/11/2018 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Sweden | |||
| 39 | EUCTR2011-002750-31-PL (EUCTR) | 03/04/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Romania;Croatia;Germany;Japan;Sweden |