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 | EUCTR2014-004804-31-DE (EUCTR) | 12/04/2016 | 06/08/2015 | A long-term study on changes in height and weight of children with MPS II receiving Elaprase and who started the treatment before the age of 6 years. | A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients with MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age | Hunter syndrome (Mucopolysaccharidosis II, [MPS II]);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Elaprase INN or Proposed INN: IDURSULFASE | Shire Human Genetic Therapies, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 4 | United States;Serbia;Philippines;Saudi Arabia;Thailand;Malaysia;Costa Rica;Oman;Dominican Republic;Germany;Vietnam | ||
| 2 | NCT02455622 (ClinicalTrials.gov) | October 28, 2015 | 20/5/2015 | Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age | A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age | Hunter Syndrome | Drug: Elaprase for intravenous (IV) infusion | Shire | NULL | Active, not recruiting | N/A | 6 Years | Male | 21 | Phase 4 | United States;Dominican Republic;Germany;Malaysia;Philippines;Serbia;Thailand;Vietnam;Oman;Saudi Arabia |
| 3 | NCT02412787 (ClinicalTrials.gov) | October 28, 2015 | 1/4/2015 | Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 | An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment | Hunter Syndrome | Drug: Idursulfase-IT;Drug: Elaprase | Shire | NULL | Active, not recruiting | N/A | 18 Years | Male | 49 | Phase 2;Phase 3 | United States;Australia;Canada;France;Mexico;Spain;United Kingdom;Argentina;Colombia |
| 4 | EUCTR2014-004143-13-ES (EUCTR) | 09/04/2015 | 09/02/2015 | An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in Patients with Hunter Syndrome and Cognitive Impairment | Hunter syndrome and cognitive impairment MedDRA version: 18.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 42 | United States;Mexico;Canada;Argentina;Spain;Colombia;United Kingdom | |||
| 5 | EUCTR2014-004143-13-GB (EUCTR) | 19/03/2015 | 30/01/2015 | An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in Patients with Hunter Syndrome and Cognitive Impairment | Hunter syndrome and cognitive impairment MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 54 | Phase 2;Phase 3 | United States;France;Mexico;Canada;Argentina;Spain;Australia;Colombia;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 |
| 6 | NCT02055118 (ClinicalTrials.gov) | March 24, 2014 | 17/1/2014 | Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment | A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment | Hunter Syndrome | Biological: idursulfase-IT;Other: No IT treatment | Shire | NULL | Completed | N/A | 18 Years | Male | 58 | Phase 2;Phase 3 | United States;Australia;Canada;France;Mexico;Spain;United Kingdom;Argentina;Colombia |
| 7 | EUCTR2013-002885-38-ES (EUCTR) | 26/11/2013 | 08/10/2013 | Peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | A Controlled,Randomized,Two-arm,Open-label,Assessor-blinded,Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction with Elaprase® in Pediatric Patients with Hunter Syndrome and early Cognitive Impairment. | Treatment of Hunter syndrome and cognitive impairment MedDRA version: 16.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASA Other descriptive name: idursulfase-IT | Shire HGT Inc | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 2;Phase 3 | United States;Mexico;Argentina;Spain;United Kingdom | ||
| 8 | EUCTR2013-002885-38-GB (EUCTR) | 22/10/2013 | 11/09/2013 | Peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | A Controlled,Randomized,Two-arm,Open-label,Assessor-blinded,Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction with Elaprase® in Pediatric Patients with Hunter Syndrome and Early Cognitive Impairment. | Long-term treatment of Hunter syndrome and cognitive impairment MedDRA version: 20.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Shire HGT Inc | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2;Phase 3 | France;United States;Mexico;Canada;Argentina;Spain;Australia;Germany;Colombia;United Kingdom | |||
| 9 | NCT01602601 (ClinicalTrials.gov) | April 9, 2012 | 17/5/2012 | A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 | A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party | Mucopolysaccharidosis II | Drug: Idursulfase;Drug: GSK2788723 | GlaxoSmithKline | NULL | Completed | N/A | N/A | All | 10 | N/A | Japan |
| 10 | EUCTR2011-000212-25-GB (EUCTR) | 05/01/2012 | 24/02/2011 | An extension to the safety, tolerability and preliminary efficacy study of Idursulfase-IT in patients with Hunter syndrome associated with learning disability | An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment | Treatment of Hunter syndrome and cognitive impairment MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase(12S)-IT Product Code: 12S-IT INN or Proposed INN: idursulfase Other descriptive name: Idursulfase-IT | Shire HGT Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 1;Phase 2 | United States;Canada;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 |
| 11 | EUCTR2010-020048-36-GB (EUCTR) | 31/01/2011 | 17/06/2010 | A safety, tolerability and preliminary efficacy study of idursulfase-IT in patients with Hunter syndrome associated with learning disability | A Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT administered in conjunction with intravenous Elaprase in Pediatric Patients with Hunter Syndrome and Cognitive Impairment | Treatment of Hunter syndrome and cognitive impairment MedDRA version: 14.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Shire HGT, Inc | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 1;Phase 2 | United States;United Kingdom | |||
| 12 | NCT01506141 (ClinicalTrials.gov) | August 1, 2010 | 15/12/2011 | Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment | An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment | Hunter Syndrome | Drug: Idursulfase-IT;Drug: Elaprase | Shire | NULL | Active, not recruiting | 3 Years | 18 Years | Male | 20 | Phase 1;Phase 2 | United States;Canada;United Kingdom |
| 13 | NCT01301898 (ClinicalTrials.gov) | May 2010 | 22/2/2011 | To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients | Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients | Mucopolysaccharidosis II | Drug: GC1111_0.5mg/kg;Drug: GC1111_1.0mg/kg;Drug: Elaprase_0.5mg/kg | Green Cross Corporation | NULL | Completed | 6 Years | 35 Years | Male | 31 | Phase 1;Phase 2 | Korea, Republic of |
| 14 | NCT00920647 (ClinicalTrials.gov) | November 1, 2009 | 12/6/2009 | A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® | A Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment | Hunter Syndrome | Other: Control;Drug: Idursulfase IT (1 mg);Drug: Idursulfase IT (10 mg);Drug: Idursulfase IT (30 mg) | Shire | NULL | Completed | 3 Years | 18 Years | Male | 16 | Phase 1;Phase 2 | United States;United Kingdom |
| 15 | EUCTR2007-001453-26-NL (EUCTR) | 02/07/2007 | 13/11/2009 | Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. | Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. | Mucopolysaccharidosis type I, II and VI. MedDRA version: 12.0;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV MedDRA version: 12.0;Classification code 10056886;Term: Mucopolysaccharidosis I MedDRA version: 12.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II | Trade Name: Elaprase Product Name: idursulfase INN or Proposed INN: IDURSULFASE Other descriptive name: Elaprase Trade Name: Naglazyme Product Name: Naglazyme INN or Proposed INN: GALSULFASE Trade Name: Aldurazyme Product Name: Aldurazyme INN or Proposed INN: LARONIDASE Other descriptive name: Aldurazyme | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Netherlands | ||||
| 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 | EUCTR2013-002885-38-FR (EUCTR) | 02/05/2016 | Peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | A Controlled,Randomized,Two-arm,Open-label,Assessor-blinded,Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction with Elaprase® in Pediatric Patients with Hunter Syndrome and Early Cognitive Impairment. | Long-term treatment of Hunter syndrome and cognitive impairment MedDRA version: 19.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT | Shire HGT Inc | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2;Phase 3 | United States;France;Mexico;Argentina;Spain;Australia;Germany;United Kingdom | |||
| 17 | EUCTR2014-004143-13-FR (EUCTR) | 16/03/2017 | An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment | An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in Patients with Hunter Syndrome and Cognitive Impairment | Hunter syndrome and cognitive impairment MedDRA version: 19.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.1;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT | Shire Human Genetic Therapies, Inc. | NULL | NA | Female: no Male: yes | 54 | Phase 2;Phase 3 | United States;France;Mexico;Canada;Argentina;Spain;Australia;Colombia;United Kingdom | |||
| 18 | EUCTR2011-000212-25-Outside-EU/EEA (EUCTR) | 19/02/2014 | An extension to the safety, tolerability and preliminary efficacy study of Idursulfase-IT in patients with Hunter syndrome associated with learning disability | An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment | Treatment of Hunter syndrome and cognitive impairment MedDRA version: 16.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Idursulfase(I2S)-IT Product Code: I2S-IT INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT | Shire HGT Inc | NULL | NA | Female: no Male: yes | 15 | Canada;United States | ||||
| 19 | EUCTR2007-006044-22-PL (EUCTR) | 21/04/2008 | A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available | A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 10.1;Level: PT;Classification code 10056889;Term: | Trade Name: Elaprase 2mg/ml Product Name: Idursulfase (I2S) Product Code: I2S INN or Proposed INN: Idursulfase Other descriptive name: Idursulfase | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: no Male: yes | 30 | Phase 4 | Poland |