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 172. Hypophosphatasia
 [ 22 clinical trials,    12 drugs(DrugBank: 5 drugs),    3 target genes / 5 target pathways

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.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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size
PhaseCountries
1EUCTR2017-001831-38-Outside-EU/EEA22 May 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-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 GmbHNot AvailableFemale: yes
Male: yes
19Phase 2United States;Canada
2EUCTR2017-003153-42-Outside-EU/EEA21 August 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 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 GmbHNot AvailableFemale: yes
Male: yes
12Phase 2United States;Canada
3NCT02797821June 6, 201630 September 2019Pharmacokinetic 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 PharmaceuticalsNot recruiting18 YearsN/AAll27Phase 2United States;Germany
4EUCTR2015-003131-35-DE19/04/201630 April 2018A 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 HypophosphatasiaHypophosphatasia 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 GmbHNot RecruitingFemale: yes
Male: yes
36Phase 2United States;Germany
5EUCTR2015-000809-39-FR04/02/201630 April 2018Access 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
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 SàrlNot RecruitingFemale: yes
Male: yes
100Phase 4France;Belgium;Spain;Russian Federation;Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02496689July 9, 20158 April 2019Expanded 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 PharmaceuticalsNot recruitingN/AN/AAllPhase 1/Phase 2United States;France
7NCT02531867June 201516 December 2017Post-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 PharmaceuticalsNot recruitingN/AN/AAll13Phase 4
8JPRN-UMIN0000148162014/08/112 April 2019A 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 CenterNot RecruitingNot applicableNot applicableMale and Female20Not selectedJapan
9NCT02235493August 201425 March 2019Non-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 PharmaceuticalsNot recruiting5 YearsN/AAll6Early Phase 1United States
10EUCTR2010-024013-31-DE30/06/20117 January 2013A 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 hypophosphatasiaHypophosphatasia (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 AGNot RecruitingFemale: yes
Male: yes
9Germany
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2010-019850-42-DE04/03/201129 May 2017An 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 GmbHNot RecruitingFemale: yes
Male: yes
100Phase 2United States;Taiwan;Saudi Arabia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Germany;Japan
12JPRN-UMIN0000038282010/07/012 April 2019Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe HypophosphatasiaHypophosphatasia1. 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 TechnologyNot RecruitingNot applicable6months-oldMale and Female5Phase 2,3Japan
13NCT01176266July 201025 March 2019Open-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 PharmaceuticalsNot recruitingN/A5 YearsAll69Phase 2/Phase 3United States;Australia;Canada;France;Germany;Italy;Japan;Russian Federation;Saudi Arabia;Spain;Turkey;United Kingdom;Taiwan
14NCT01163149June 201025 March 2019Safety 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 PharmaceuticalsNot recruiting13 Years65 YearsAll19Phase 2United States;Canada
15NCT01203826April 201025 March 2019Extension 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 PharmaceuticalsNot recruiting5 Years12 YearsAll12Phase 2United States;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00952484September 20098 April 2019Safety 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 PharmaceuticalsNot recruiting5 Years12 YearsAll13Phase 2United States;Canada
17NCT00894075July 200919 February 2015Safety 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 SarlNot recruiting5 Years12 YearsBoth0Phase 2United States
18NCT01205152April 200925 March 2019Extension 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 PharmaceuticalsNot recruiting24 Weeks42 MonthsAll10Phase 2United States;United Arab Emirates;United Kingdom
19EUCTR2009-009369-32-GB30/03/200930 April 2019Extension 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
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 GmbHNot Recruiting Female: yes
Male: yes
10Phase 2United States;United Arab Emirates;United Kingdom
20EUCTR2008-007406-11-GB25/02/200920 March 2012A 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 PartnershipNot RecruitingFemale: yes
Male: yes
10United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT00744042September 20088 April 2019Safety 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 PharmaceuticalsNot recruitingN/A36 MonthsAll11Phase 1/Phase 2United States;Canada;United Arab Emirates;United Kingdom
22NCT00739505August 20088 April 2019Safety 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 PharmaceuticalsNot recruiting18 Years80 YearsAll6Phase 1United States;Canada

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