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 | NCT04980248 (ClinicalTrials.gov) | July 22, 2021 | 23/7/2021 | Study of ALXN1850 in Participants With Hypophosphatasia (HPP) | A Phase 1, Open-label, Dose-escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALXN1850 in Adults With Hypophosphatasia | Hypophosphatasia | Biological: ALXN1850 | Alexion Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 15 | Phase 1 | United States |
| 3 | 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With Pediatric-Onset Hypophosphatasia | Hypophosphatasia | Drug: asfotase alfa | Alexion Pharmaceuticals | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 4 | NULL |
| 4 | 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 | |
| 5 | NCT04181164 (ClinicalTrials.gov) | October 1, 2019 | 18/11/2019 | Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP) | Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | Other: Microindentation;Other: High resolution peripheral quantitative computed tomography (HRpQCT);Biological: Biochemical analysis of different bone markers. | Hvidovre University Hospital | Odense University Hospital | Active, not recruiting | 18 Years | N/A | All | 30 | Denmark | |
| 6 | 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 |
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 |
| 11 | 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 | |
| 12 | 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 |
| 13 | NCT02235493 (ClinicalTrials.gov) | August 2014 | 5/9/2014 | 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 | NULL | Completed | 5 Years | N/A | All | 6 | United States | |
| 14 | 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 | ||
| 15 | 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 | ||
| 16 | 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 | ||
| 17 | NCT01406977 (ClinicalTrials.gov) | July 2011 | 29/7/2011 | Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP) | 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 (HPP). | Hypophosphatasia | Drug: BPS804 | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 70 Years | Both | 9 | Phase 2 | Germany |
| 18 | EUCTR2010-024013-31-DE (EUCTR) | 30/06/2011 | 07/02/2011 | 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 Other descriptive name: fully human IgG2 lambda monoclonal antibody | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 9 | Germany | |||
| 19 | 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 | ||
| 20 | JPRN-UMIN000003828 | 2010/07/01 | 01/07/2010 | 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 | Complete: follow-up complete | Not applicable | 6months-old | Male and Female | 5 | Phase 2,3 | Japan | |
| 21 | 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 |
| 22 | 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 |
| 23 | 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 |
| 24 | 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 |
| 25 | NCT00894075 (ClinicalTrials.gov) | July 2009 | 4/5/2009 | 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 | NULL | Withdrawn | 5 Years | 12 Years | Both | 0 | Phase 2 | United States |
| 26 | 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 |
| 27 | 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 | ||
| 28 | EUCTR2008-007406-11-GB (EUCTR) | 25/02/2009 | 15/01/2009 | 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 Other descriptive name: Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein | Enobia Canada Limited Partnership | NULL | Not Recruiting | Female: yes Male: yes | 10 | United Kingdom | |||
| 29 | 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 |
| 30 | 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 |
| 31 | 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 | |||
| 32 | 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 | |||
| 33 | 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 | ||||
| 34 | 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 |