172. 低ホスファターゼ症 Hypophosphatasia Clinical trials / Disease details
臨床試験数 : 34 / 薬物数 : 19 - (DrugBank : 4) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 6
| 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 | NCT05234567 (ClinicalTrials.gov) | February 17, 2022 | 1/2/2022 | A Prospective Sub-Study of the Global Hypophosphatasia Registry | A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia | Hypophosphatasia | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Not yet recruiting | N/A | N/A | All | 40 | United States | |
| 2 | NCT04189315 (ClinicalTrials.gov) | April 2020 | 4/12/2019 | Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease | A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia | Hypophosphatasia | Drug: asfotase alfa | Alexion Pharmaceuticals | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 4 | NULL |
| 3 | 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 | |
| 4 | NCT02797821 (ClinicalTrials.gov) | June 6, 2016 | 23/5/2016 | Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP) | A Phase 2a, Randomized, Multicenter, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia | Hypophosphatasia | Drug: Asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 27 | Phase 2 | United States;Germany |
| 5 | NCT02496689 (ClinicalTrials.gov) | May 17, 2016 | 8/7/2015 | Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP) | 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 | Biological: asfotase alfa | Alexion Pharmaceuticals | NULL | Approved for marketing | N/A | N/A | All | United States;France | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | NCT02531867 (ClinicalTrials.gov) | June 2015 | 11/8/2015 | Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan | A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in Japan | Hypophosphatasia | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Completed | N/A | N/A | All | 13 | Phase 4 | NULL |
| 9 | JPRN-UMIN000014816 | 2014/08/11 | 11/08/2014 | A Multicenter study of the safety and efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in patients with hypophosphatasia (HPP) | hypophosphatasia | Drug: Asfotase Alfa (ALXN1215) Dose: 6mg/kg/week (divided 3 times a week) | HPP study group | Translational Research Informatics Center | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 20 | Not selected | Japan | |
| 10 | NCT02456038 (ClinicalTrials.gov) | August 2014 | 26/3/2015 | Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP) | A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophosphatasia (HPP) | Hypophosphatasia | Drug: Asfotase Alfa (ALXN1215) | Translational Research Informatics Center, Kobe, Hyogo, Japan | Osaka University Graduate School of Medicine | Completed | N/A | N/A | Both | 13 | Phase 2 | Japan |
| 11 | EUCTR2010-019850-42-GB (EUCTR) | 05/09/2013 | 01/02/2013 | 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 MedDRA version: 19.1;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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Taiwan;Saudi Arabia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Germany;Japan | ||
| 12 | EUCTR2010-019850-42-ES (EUCTR) | 28/06/2013 | 09/04/2013 | 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 MedDRA version: 14.1;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Asfotase alfa Product Code: ENB-0040 INN or Proposed INN: Asfotase alfa Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Product Name: Asfotase 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 | 60 | Phase 2 | United States;Saudi Arabia;Taiwan;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Australia;Germany;Japan | ||
| 13 | EUCTR2010-019850-42-IT (EUCTR) | 09/05/2013 | 01/03/2013 | 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 MedDRA version: 15.1;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Asfotase alfa Product Code: ENB-0040 INN or Proposed INN: Asfotase alfa Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Product Name: Asfotase 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 | 60 | Phase 2 | United States;Saudi Arabia;Taiwan;Spain;Turkey;Italy;United Kingdom;France;Canada;Argentina;Australia;Germany;Japan | ||
| 14 | 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 | ||
| 15 | NCT01176266 (ClinicalTrials.gov) | July 2010 | 29/7/2010 | Open-Label Study of Asfotase Alfa 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 | Drug: asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | N/A | 5 Years | All | 69 | Phase 2/Phase 3 | United States;Australia;Canada;France;Germany;Italy;Japan;Russian Federation;Saudi Arabia;Spain;Turkey;United Kingdom;Taiwan |
| 16 | NCT01163149 (ClinicalTrials.gov) | June 2010 | 24/6/2010 | 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, Pharmacokinetic of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Adolescents and Adults With Hypophosphatasia (HPP) | Hypophosphatasia | Drug: asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | 13 Years | 65 Years | All | 19 | Phase 2 | United States;Canada |
| 17 | NCT01203826 (ClinicalTrials.gov) | April 2010 | 15/9/2010 | Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) | Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Completed | 5 Years | 12 Years | All | 12 | Phase 2 | United States;Canada |
| 18 | NCT00952484 (ClinicalTrials.gov) | September 2009 | 3/8/2009 | Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) | A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Historical Control Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | Biological: asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | 5 Years | 12 Years | All | 13 | Phase 2 | United States;Canada |
| 19 | NCT01205152 (ClinicalTrials.gov) | April 2009 | 17/9/2010 | Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP) | Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children With Hypophosphatasia (HPP) | Hypophosphatasia | Biological: asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | 24 Weeks | 42 Months | All | 10 | Phase 2 | United States;United Arab Emirates;United Kingdom |
| 20 | EUCTR2009-009369-32-GB (EUCTR) | 30/03/2009 | 14/04/2009 | Extension open-label use of ENB-0040 for infantile hypophosphatasia | Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children with Hypophosphatasia (HPP) | Infantile hypoposphatasia MedDRA version: 19.1;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Strensiq Product Name: Asfotase alfa Product Code: 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 | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Arab Emirates;United Kingdom | ||
| 21 | NCT00744042 (ClinicalTrials.gov) | September 2008 | 27/8/2008 | Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) | A Multicenter, Open-Label Study of the Safety, Tolerability and Pharmacology of Asfotase Alfa in up to 10 Severely Affected Patients With for the Treatment of Severely Affected Patients With Infantile Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | Biological: asfotase alfa | Alexion Pharmaceuticals | NULL | Completed | N/A | 36 Months | All | 11 | Phase 1/Phase 2 | United States;Canada;United Arab Emirates;United Kingdom |
| 22 | NCT00739505 (ClinicalTrials.gov) | August 2008 | 19/8/2008 | Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) | A Multicenter, Open-Label, Dose Escalating Study of the Safety, Tolerability and Pharmacology of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Completed | 18 Years | 80 Years | All | 6 | Phase 1 | United States;Canada |
| 23 | 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 | |||
| 24 | EUCTR2015-001128-52-Outside-EU/EEA (EUCTR) | 28/05/2015 | Safety and efficacy of asfotase alfa in children with hypophosphatasia | A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Historical Control Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Children with Hypophosphatasia (HPP) | Hypophosphatasia (HPP) 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] | Product Name: Asfotase Alfa INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein | Alexion Pharma International Sàrl | NULL | NA | Female: yes Male: yes | 13 | United States;Canada | ||||
| 25 | 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 | |||
| 26 | EUCTR2010-019850-42-FR (EUCTR) | 28/09/2015 | 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 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] | Product Name: Asfotase alfa Product Code: ENB-0040 INN or Proposed INN: Asfotase alfa Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Product Name: Asfotase 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 | NA | Female: yes Male: yes | 60 | Phase 2 | United States;Taiwan;Saudi Arabia;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Australia;Germany;Japan |