19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2014-003960-20-IT (EUCTR) | 16/09/2015 | 05/11/2020 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Mucopolysaccharidosis Type IIIA Disease - NA | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan Nsulfatase (rhHNS) Product Code: [HGT-1410] INN or Proposed INN: NA Other descriptive name: Recombinant human heparan N-sulfatase | SHIRE HUMAN GENETIC THERAPIES, INC | NULL | Not Recruiting | Female: yes Male: yes | 17 | Phase 2 | United States;France;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2014-003960-20-NL (EUCTR) | 18/08/2015 | 05/03/2015 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | An Open-Label Extension of Study HGT-SAN-093 Evaluating theSafety and Efficacy Study of HGT-1410 (Recombinant HumanHeparan N Sulfatase) Administration via an Intrathecal DrugDelivery Device in Pediatric Patients with MucopolysaccharidosisType IIIA Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 17 | Phase 2 | United States;France;Spain;Germany;Netherlands;United Kingdom;Italy | ||
| 3 | EUCTR2014-003960-20-GB (EUCTR) | 10/08/2015 | 01/04/2015 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Mucopolysaccharidosis Type IIIA Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 19.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 17 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2014-003960-20-DE (EUCTR) | 15/06/2015 | 26/01/2015 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | An Open-Label Extension of Study HGT-SAN-093 Evaluating theSafety and Efficacy Study of HGT-1410 (Recombinant HumanHeparan N Sulfatase) Administration via an Intrathecal DrugDelivery Device in Pediatric Patients with MucopolysaccharidosisType IIIA Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 17 | Phase 2 | France;United States;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 5 | NCT02350816 (ClinicalTrials.gov) | April 8, 2015 | 21/1/2015 | An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. | An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Mucopolysaccharidosis Type IIIA Disease | Sanfilippo Syndrome;Mucopolysaccharidosis (MPS) | Drug: HGT-1410 | Shire | NULL | Terminated | 12 Months | 48 Months | All | 17 | Phase 2 | United States;France;Germany;Italy;Netherlands;Spain;United Kingdom |
| 6 | EUCTR2014-003960-20-ES (EUCTR) | 18/02/2015 | 26/01/2015 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | A Open-Label Extension of Study HGT-SAN-093 Evaluating theSafety and Efficacy Study of HGT-1410 (Recombinant HumanHeparan N Sulfatase) Administration via an Intrathecal DrugDelivery Device in Pediatric Patients with MucopolysaccharidosisType IIIA Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 17.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;United States;Argentina;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 7 | EUCTR2013-003450-24-IT (EUCTR) | 11/09/2014 | 09/05/2014 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 17.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 8 | EUCTR2013-003450-24-DE (EUCTR) | 09/04/2014 | 07/11/2013 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 17.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;United States;Argentina;Brazil;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 9 | EUCTR2013-003450-24-NL (EUCTR) | 03/03/2014 | 21/11/2013 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 17.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;United States;Argentina;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 10 | EUCTR2013-003450-24-ES (EUCTR) | 16/01/2014 | 12/11/2013 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 14.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: heparán N sulfatasa humana recombinante (rhHNS) Product Code: HGT-1410 INN or Proposed INN: No disponible Other descriptive name: Heparán N sulfatasa humana recombinante | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;United States;Argentina;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 11 | EUCTR2013-003450-24-GB (EUCTR) | 08/01/2014 | 08/11/2013 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 19.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Argentina;Brazil;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 12 | EUCTR2010-021348-16-NL (EUCTR) | 17/02/2011 | 09/02/2011 | Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients | An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients with Sanfilippo Syndrome Type A (MPS IIIA) | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 19.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | Netherlands;United Kingdom | ||
| 13 | EUCTR2010-021348-16-GB (EUCTR) | 09/02/2011 | 20/12/2010 | Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients with Sanfilippo Syndrome Type A (MPS IIIA) | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 19.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | Not Recruiting | Female: yes Male: yes | 7 | Phase 1;Phase 2 | Netherlands;United Kingdom | ||
| 14 | EUCTR2009-015984-15-GB (EUCTR) | 25/03/2010 | 17/03/2010 | A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 14.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;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 heparan N-sulfatase (rhHNS) Product Code: HGT-1410 Other descriptive name: Recombinant human heparan N-sulfatase (rhHNS) | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 15 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 15 | EUCTR2013-003450-24-FR (EUCTR) | 29/09/2015 | Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A | A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 18.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire Human Genetic Therapies, Inc | NULL | NA | Female: yes Male: yes | 18 | Phase 2 | United States;France;Argentina;Brazil;Spain;Netherlands;Germany;Italy;United Kingdom | |||
| 16 | EUCTR2014-003960-20-FR (EUCTR) | 25/06/2015 | An Extension Study to Evaluate the Safety and Efficacy of HGT-1410 Administration in Pediatric Patients with Sanfilippo Syndrome Type A | A Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Mucopolysaccharidosis Type IIIA Disease | Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA) MedDRA version: 18.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human heparan N-sulfatase (rhHNS) Product Code: HGT-1410 INN or Proposed INN: Not available Other descriptive name: Recombinant human heparan N-sulfatase | Shire human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Argentina;Spain;Netherlands;Germany;Italy;United Kingdom |