19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | 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 |
| 2 | 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 | |||
| 3 | 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 | ||
| 4 | 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 |
| 5 | 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 | ||
| 6 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Mexico;Canada;Argentina;Spain;Australia;Germany;Colombia;United Kingdom | ||
| 7 | 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 | ||
| 8 | 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] | Product Name: Idursulfase (I2S)-IT Product Code: I2S-IT INN or Proposed INN: NA Other descriptive name: Idursulfase-IT | Shire HGT, Inc | NULL | Not Recruiting | Female: no Male: yes | 20 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 9 | 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 | 25 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 10 | NCT00920647 (ClinicalTrials.gov) | November 18, 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 |
| 11 | 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 | |||
| 12 | 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 | ||||
| 13 | 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 |