172. Hypophosphatasia
32 clinical trials, 18 drugs (DrugBank: 4 drugs), 3 drug target genes, 6 drug target pathways
Searched query = "Hypophosphatasia"
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 | NCT04195763 (ClinicalTrials.gov) | November 6, 2019 | 10/12/2019 | Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa) | A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia | Hypophosphatasia | Drug: asfotase alfa | Alexion Pharmaceuticals | Xcenda, LLC | Enrolling by invitation | 18 Years | N/A | All | 100 | United States | |
| 2 | EUCTR2015-003131-35-DE (EUCTR) | 19/04/2016 | 03/12/2015 | A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia | A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia | Hypophosphatasia is a rare inborn error of bone metabolism caused by inactivating mutations in the gene encoding the Tissue-nonspecific alkaline phosphatase isoenzyme.With deficiency of Tissue-nonspecific alkaline phosphatase, there is a buildup of extracellular inorganic pyrophosphate, which inhibits mineralization of bone matrix. MedDRA version: 20.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Strensiq Product Name: asfotase alfa INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein | Alexion Pharma GmbH | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 2 | United States;Germany | ||
| 3 | EUCTR2015-000809-39-FR (EUCTR) | 04/02/2016 | 05/08/2015 | Access Program for Asfotase Alfa | An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (human recombinant tissue-nonspecific alkaline phosphatase fusion protein) Treatment for Patients with Infantile- or Juvenile-onset Hypophosphatasia (HPP) | Hypophosphatasia MedDRA version: 18.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Strensiq Product Name: Asfotas Alfa Product Code: ENB-0040 INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein | Alexion Pharma International Sàrl | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 4 | France;Belgium;Spain;Russian Federation;Netherlands | ||
| 4 | EUCTR2010-019850-42-DE (EUCTR) | 04/03/2011 | 15/10/2010 | An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children =5 Years of Age with Hypophosphatasia (HPP) | An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children =5 Years of Age with Hypophosphatasia (HPP) | Hypophosphatasia is a rare inborn error of bone metabolism caused by inactivating mutations in the gene encoding the Tissue-nonspecific alkaline phosphatase isoenzyme. With deficiency of Tissue-nonspecific alkaline phosphatase, there is a buildup of extracellular inorganic pyrophosphate, which inhibits mineralization of bone matrix. MedDRA version: 20.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Strensiq Product Name: Asfotase alfa INN or Proposed INN: Asfotase alfa Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Trade Name: Strensiq Product Name: Asfotase alfa INN or Proposed INN: Asfotase alfa Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein | Alexion Pharma GmbH | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 2 | United States;Taiwan;Saudi Arabia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Germany;Japan | ||
| 5 | EUCTR2017-003153-42-Outside-EU/EEA (EUCTR) | 03/08/2017 | Long-Term Safety and Efficacy of Asfotase Alfa in Children with Hypophosphatasia (HPP) | Extension Study of Protocol ENB-006-09 Evaluating the Long-Term Safety and Efficacy ofAsfotase Alfa (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Children with Hypophosphatasia (HPP) | Hypophosphatasia MedDRA version: 20.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Strensiq Product Name: Asfotase alfa INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein | Alexion Pharma GmbH | NULL | NA | Female: yes Male: yes | 12 | Phase 2 | United States;Canada | |||
| 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 | EUCTR2017-001831-38-Outside-EU/EEA (EUCTR) | 09/05/2017 | Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP) | A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Concurrent Control Study of the Safety, Efficacy, and Pharmacokinetics of ENB-0040 (Human Recombinant Tissue-NonspecificAlkaline Phosphatase Fusion Protein) in Adolescents and Adults with Hypophosphatasia (HPP) | Hypophosphatasia MedDRA version: 20.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Strensiq Product Name: Asfotase alfa INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein | Alexion Pharma GmbH | NULL | NA | Female: yes Male: yes | 19 | Phase 2 | United States;Canada |