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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05234567
(ClinicalTrials.gov)
February 17, 20221/2/2022A Prospective Sub-Study of the Global Hypophosphatasia RegistryA 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 HypophosphatasiaHypophosphatasiaBiological: Asfotase AlfaAlexion PharmaceuticalsNULLNot yet recruitingN/AN/AAll40United States
2NCT04980248
(ClinicalTrials.gov)
July 22, 202123/7/2021Study 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 HypophosphatasiaHypophosphatasiaBiological: ALXN1850Alexion PharmaceuticalsNULLRecruiting18 YearsN/AAll15Phase 1United States
3NCT04189315
(ClinicalTrials.gov)
April 20204/12/2019Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic DiseaseA 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 HypophosphatasiaHypophosphatasiaDrug: asfotase alfaAlexion PharmaceuticalsNULLWithdrawn18 YearsN/AAll0Phase 4NULL
4NCT04195763
(ClinicalTrials.gov)
November 6, 201910/12/2019Patient 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 HypophosphatasiaHypophosphatasiaDrug: asfotase alfaAlexion PharmaceuticalsXcenda, LLCEnrolling by invitation18 YearsN/AAll100United States
5NCT04181164
(ClinicalTrials.gov)
October 1, 201918/11/2019Evaluation 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 HospitalOdense University HospitalActive, not recruiting18 YearsN/AAll30Denmark
6NCT02797821
(ClinicalTrials.gov)
June 6, 201623/5/2016Pharmacokinetic 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 HypophosphatasiaHypophosphatasiaDrug: Asfotase alfaAlexion PharmaceuticalsNULLCompleted18 YearsN/AAll27Phase 2United States;Germany
7NCT02496689
(ClinicalTrials.gov)
May 17, 20168/7/2015Expanded 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)HypophosphatasiaBiological: asfotase alfaAlexion PharmaceuticalsNULLApproved for marketingN/AN/AAllUnited States;France
8EUCTR2015-003131-35-DE
(EUCTR)
19/04/201603/12/2015A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset HypophosphatasiaA 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
36Phase 2United States;Germany
9EUCTR2015-000809-39-FR
(EUCTR)
04/02/201605/08/2015Access Program for Asfotase AlfaAn 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 SàrlNULLNot RecruitingFemale: yes
Male: yes
100Phase 4France;Belgium;Spain;Russian Federation;Netherlands
10NCT02531867
(ClinicalTrials.gov)
June 201511/8/2015Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in JapanA Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in JapanHypophosphatasiaBiological: Asfotase AlfaAlexion PharmaceuticalsNULLCompletedN/AN/AAll13Phase 4NULL
11JPRN-UMIN000014816
2014/08/1111/08/2014A Multicenter study of the safety and efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in patients with hypophosphatasia (HPP) hypophosphatasiaDrug: Asfotase Alfa (ALXN1215)
Dose: 6mg/kg/week (divided 3 times a week)
HPP study groupTranslational Research Informatics CenterComplete: follow-up completeNot applicableNot applicableMale and Female20Not selectedJapan
12NCT02456038
(ClinicalTrials.gov)
August 201426/3/2015Safety 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)HypophosphatasiaDrug: Asfotase Alfa (ALXN1215)Translational Research Informatics Center, Kobe, Hyogo, JapanOsaka University Graduate School of MedicineCompletedN/AN/ABoth13Phase 2Japan
13NCT02235493
(ClinicalTrials.gov)
August 20145/9/2014Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09A Single-Center, Non-interventional Substudy of ALX-HPP-502 to Assess Functional Natural History Data of Patients With Juvenile Historical Controls in ENB-006-09Hypophosphatasia;Bone DiseaseBiological: Retrospective Case OnlyAlexion PharmaceuticalsNULLCompleted5 YearsN/AAll6United States
14EUCTR2010-019850-42-GB
(EUCTR)
05/09/201301/02/2013An 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 GmbHNULLNot 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): noUnited States;Taiwan;Saudi Arabia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Germany;Japan
15EUCTR2010-019850-42-ES
(EUCTR)
28/06/201309/04/2013An 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 SàrlNULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Saudi Arabia;Taiwan;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Australia;Germany;Japan
16EUCTR2010-019850-42-IT
(EUCTR)
09/05/201301/03/2013An 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 SàrlNULLNot RecruitingFemale: yes
Male: yes
60Phase 2United States;Saudi Arabia;Taiwan;Spain;Turkey;Italy;United Kingdom;France;Canada;Argentina;Australia;Germany;Japan
17NCT01406977
(ClinicalTrials.gov)
July 201129/7/2011Dose 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).HypophosphatasiaDrug: BPS804Novartis PharmaceuticalsNULLCompleted18 Years70 YearsBoth9Phase 2Germany
18EUCTR2010-024013-31-DE
(EUCTR)
30/06/201107/02/2011A clinical study to evaluate the safety and preliminary efficacy of BPS804 in adults with hypophosphatasiaAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
9Germany
19EUCTR2010-019850-42-DE
(EUCTR)
04/03/201115/10/2010An 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 GmbHNULLNot RecruitingFemale: yes
Male: yes
100Phase 2United States;Taiwan;Saudi Arabia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Germany;Japan
20JPRN-UMIN000003828
2010/07/0101/07/2010Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia Hypophosphatasia1. 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 MedicineNational Institute of Advanced Industrial Science and TechnologyComplete: follow-up completeNot applicable6months-oldMale and Female5Phase 2,3Japan
21NCT01176266
(ClinicalTrials.gov)
July 201029/7/2010Open-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)HypophosphatasiaDrug: asfotase alfaAlexion PharmaceuticalsNULLCompletedN/A5 YearsAll69Phase 2/Phase 3United States;Australia;Canada;France;Germany;Italy;Japan;Russian Federation;Saudi Arabia;Spain;Turkey;United Kingdom;Taiwan
22NCT01163149
(ClinicalTrials.gov)
June 201024/6/2010Safety 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)HypophosphatasiaDrug: asfotase alfaAlexion PharmaceuticalsNULLCompleted13 Years65 YearsAll19Phase 2United States;Canada
23NCT01203826
(ClinicalTrials.gov)
April 201015/9/2010Extension 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 AlfaAlexion PharmaceuticalsNULLCompleted5 Years12 YearsAll12Phase 2United States;Canada
24NCT00952484
(ClinicalTrials.gov)
September 20093/8/2009Safety 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 alfaAlexion PharmaceuticalsNULLCompleted5 Years12 YearsAll13Phase 2United States;Canada
25NCT00894075
(ClinicalTrials.gov)
July 20094/5/2009Safety 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 ChildrenHypophosphatasiaBiological: ENB-0040Alexion Pharma International SarlNULLWithdrawn5 Years12 YearsBoth0Phase 2United States
26NCT01205152
(ClinicalTrials.gov)
April 200917/9/2010Extension 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)HypophosphatasiaBiological: asfotase alfaAlexion PharmaceuticalsNULLCompleted24 Weeks42 MonthsAll10Phase 2United States;United Arab Emirates;United Kingdom
27EUCTR2009-009369-32-GB
(EUCTR)
30/03/200914/04/2009Extension open-label use of ENB-0040 for infantile hypophosphatasiaExtension 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 GmbHNULLNot 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): noUnited States;United Arab Emirates;United Kingdom
28EUCTR2008-007406-11-GB
(EUCTR)
25/02/200915/01/2009A 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 PartnershipNULLNot RecruitingFemale: yes
Male: yes
10United Kingdom
29NCT00744042
(ClinicalTrials.gov)
September 200827/8/2008Safety 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 alfaAlexion PharmaceuticalsNULLCompletedN/A36 MonthsAll11Phase 1/Phase 2United States;Canada;United Arab Emirates;United Kingdom
30NCT00739505
(ClinicalTrials.gov)
August 200819/8/2008Safety 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 AlfaAlexion PharmaceuticalsNULLCompleted18 Years80 YearsAll6Phase 1United States;Canada
31EUCTR2017-003153-42-Outside-EU/EEA
(EUCTR)
03/08/2017Long-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 GmbHNULLNAFemale: yes
Male: yes
12Phase 2United States;Canada
32EUCTR2010-019850-42-FR
(EUCTR)
28/09/2015An 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 SàrlNULLNAFemale: yes
Male: yes
60Phase 2United States;Taiwan;Saudi Arabia;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Australia;Germany;Japan
33EUCTR2015-001128-52-Outside-EU/EEA
(EUCTR)
28/05/2015Safety and efficacy of asfotase alfa in children with hypophosphatasiaA 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 SàrlNULLNAFemale: yes
Male: yes
13United States;Canada
34EUCTR2017-001831-38-Outside-EU/EEA
(EUCTR)
09/05/2017Safety 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 GmbHNULLNAFemale: yes
Male: yes
19Phase 2United States;Canada