172. 低ホスファターゼ症 ［臨床試験数：22，薬物数：12（DrugBank：5），標的遺伝子数：3，標的パスウェイ数：5］
Searched query = "Hypophosphatasia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2017-001831-38-Outside-EU/EEA | 22 May 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-Nonspecific Alkaline 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Alexion Pharma GmbH | Not Available | Female: yes Male: yes | 19 | Phase 2 | United States;Canada | ||||
| 2 | EUCTR2017-003153-42-Outside-EU/EEA | 21 August 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 of Asfotase 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Alexion Pharma GmbH | Not Available | Female: yes Male: yes | 12 | Phase 2 | United States;Canada | ||||
| 3 | NCT02797821 | June 6, 2016 | 30 September 2019 | 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 | Not recruiting | 18 Years | N/A | All | 27 | Phase 2 | United States;Germany | |
| 4 | EUCTR2015-003131-35-DE | 19/04/2016 | 30 April 2018 | 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Alexion Pharma GmbH | Not Recruiting | Female: yes Male: yes | 36 | Phase 2 | United States;Germany | |||
| 5 | EUCTR2015-000809-39-FR | 04/02/2016 | 30 April 2018 | 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: ASFOTASE ALFA Current Sponsor code: ENB-0040 Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: range Concentration number: 40-100 | Alexion Pharma International Sàrl | Not Recruiting | Female: yes Male: yes | 100 | Phase 4 | France;Belgium;Spain;Russian Federation;Netherlands | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02496689 | July 9, 2015 | 8 April 2019 | 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 | Not recruiting | N/A | N/A | All | Phase 1/Phase 2 | United States;France | ||
| 7 | NCT02531867 | June 2015 | 16 December 2017 | 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 | Not recruiting | N/A | N/A | All | 13 | Phase 4 | ||
| 8 | JPRN-UMIN000014816 | 2014/08/11 | 2 April 2019 | 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 | Not Recruiting | Not applicable | Not applicable | Male and Female | 20 | Not selected | Japan | |
| 9 | NCT02235493 | August 2014 | 25 March 2019 | Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09 | A Single-Center, Non-interventional Substudy of ALX-HPP-502 to Assess Functional Natural History Data of Patients With Juvenile Historical Controls in ENB-006-09 | Hypophosphatasia;Bone Disease | Biological: Retrospective Case Only | Alexion Pharmaceuticals | Not recruiting | 5 Years | N/A | All | 6 | Early Phase 1 | United States | |
| 10 | EUCTR2010-024013-31-DE | 30/06/2011 | 7 January 2013 | A clinical study to evaluate the safety and preliminary efficacy of BPS804 in adults with hypophosphatasia | An open-label, intra-patient dose-escalation study to evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of multiple infusions of BPS804 in adults with hypophosphatasia | Hypophosphatasia (HPP) is a rare genetic metabolic disorder which results in impaired skeletal mineralization, and which is caused by the absence of or by deficient enzymatic activity of the tissue-nonspecific alkaline phosphatase (TNSALP) MedDRA version: 14.1 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BPS804 Product Code: BPS804 Pharmaceutical Form: Powder and solvent for solution for infusion Current Sponsor code: BPS804 Other descriptive name: fully human IgG2 lambda monoclonal antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 150- | Novartis Pharma Services AG | Not Recruiting | Female: yes Male: yes | 9 | Germany | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2010-019850-42-DE | 04/03/2011 | 29 May 2017 | 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: Asfotase alfa Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- Trade Name: Strensiq Product Name: Asfotase alfa Pharmaceutical Form: Solution for injection INN or Proposed INN: Asfotase alfa Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Alexion Pharma GmbH | 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 | |||
| 12 | JPRN-UMIN000003828 | 2010/07/01 | 2 April 2019 | Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia | Hypophosphatasia | 1. BM harvest 1)Agreement about Harvesting BM 2)Harvest volume; 100-120ml 3)Anesthetic protocol; discuss by donor 2.BMT and MSCT First, we perform allogeneic BMT, and then allogeneic MSCT from same BM donor. Only MSCT is repeatedly performed if the patient deteriorates symptoms after BMT and MSCT. 1)BMT -Timing of BMT as soon as possible after diagnosis -Conditioning Buslfan 0.9-1mg/kg/dose every 6 hours, 4days Cyclophosphamide 50mg/kg/dose, 4days Antithymocyte globulin 1.25mg/kg/dose, 4days -Prophylaxis of GVHD Methotrexate 10-15mg/m2/dose, 4days Taclolimus, 0.02-0.04mg/kg/day, about 6 months 2)MSCT -Cultured-Expansion of MSCs from BM -Preservation of MSCs -Timing of MSCT About 14 to 21 days After BMT -Administration route and volume More than 106/kg is intravenously injected. -Administration of immunosuppressant drugs -Taclolimus 0.02-0.04mg/kg/day, about 6 months 3. Examination period From July 1, 2010 to March 31, 2013 | Shimane University School of Medicine | National Institute of Advanced Industrial Science and Technology | Not Recruiting | Not applicable | 6months-old | Male and Female | 5 | Phase 2,3 | Japan | |
| 13 | NCT01176266 | July 2010 | 25 March 2019 | 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 | Not recruiting | 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 | |
| 14 | NCT01163149 | June 2010 | 25 March 2019 | 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 | Not recruiting | 13 Years | 65 Years | All | 19 | Phase 2 | United States;Canada | |
| 15 | NCT01203826 | April 2010 | 25 March 2019 | 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 | Not recruiting | 5 Years | 12 Years | All | 12 | Phase 2 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT00952484 | September 2009 | 8 April 2019 | 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 | Not recruiting | 5 Years | 12 Years | All | 13 | Phase 2 | United States;Canada | |
| 17 | NCT00894075 | July 2009 | 19 February 2015 | Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP) | Single-Center, Case-Control Study of Safety, Efficacy and Pharmacokinetics of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) for Treatment of Hypophosphatasia in Children | Hypophosphatasia | Biological: ENB-0040 | Alexion Pharma International Sarl | Not recruiting | 5 Years | 12 Years | Both | 0 | Phase 2 | United States | |
| 18 | NCT01205152 | April 2009 | 25 March 2019 | 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 | Not recruiting | 24 Weeks | 42 Months | All | 10 | Phase 2 | United States;United Arab Emirates;United Kingdom | |
| 19 | EUCTR2009-009369-32-GB | 30/03/2009 | 30 April 2019 | 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: Asfotase alfa Current Sponsor code: Asfotase alfa Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | Alexion Pharma GmbH | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;United Arab Emirates;United Kingdom | |||
| 20 | EUCTR2008-007406-11-GB | 25/02/2009 | 20 March 2012 | A Multicenter, Open-Label Study of the Safety, Tolerability and Pharmacology of ENB-0040 (Enobia’s human recombinant tissue non-specific alkaline phosphatase fusion protein) in up to 6 Severely Affected Patients with Infantile Hypophosphatasia (HPP) | A Multicenter, Open-Label Study of the Safety, Tolerability and Pharmacology of ENB-0040 (Enobia’s human recombinant tissue non-specific alkaline phosphatase fusion protein) in up to 6 Severely Affected Patients with Infantile Hypophosphatasia (HPP) | Infantile Hypophosphatasia MedDRA version: 14.1 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: ENB-0040 Pharmaceutical Form: Solution for injection Current Sponsor code: ENB-0040 Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | Enobia Canada Limited Partnership | Not Recruiting | Female: yes Male: yes | 10 | United Kingdom | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT00744042 | September 2008 | 8 April 2019 | 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 | Not recruiting | N/A | 36 Months | All | 11 | Phase 1/Phase 2 | United States;Canada;United Arab Emirates;United Kingdom | |
| 22 | NCT00739505 | August 2008 | 8 April 2019 | 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 | Not recruiting | 18 Years | 80 Years | All | 6 | Phase 1 | United States;Canada |