172. Hypophosphatasia Clinical trials / Disease details
Clinical trials : 34 / Drugs : 17 - (DrugBank : 4) / Drug target genes : 3 - Drug target pathways : 6
Showing 1 to 10 of 34 diseases
| 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) | March 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 A Prospective Observational Sub-Study of the Global HypophosphatasiaRegistry to Describe the Potenti ... | Hypophosphatasia | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Recruiting | N/A | N/A | All | 40 | United States | |
| 2 | NCT05062629 (ClinicalTrials.gov) | August 24, 2021 | 21/9/2021 | United States Hypophosphatasia Molecular Research Center | United States Hypophosphatasia Molecular Research Center | Hypophosphatasia | Genetic: Whole Genome Sequencing | Children's Mercy Hospital Kansas City | NULL | Recruiting | 1 Month | 120 Years | All | 66 | United States | |
| 3 | NCT04980248 (ClinicalTrials.gov) | August 16, 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 A Phase 1, Open-label, Dose-escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, ... | Hypophosphatasia | Biological: ALXN1850 | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 15 | Phase 1 | United States |
| 4 | 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 A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, ... | Hypophosphatasia | Drug: asfotase alfa | Alexion Pharmaceuticals | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 4 | NULL |
| 5 | NCT04195763 (ClinicalTrials.gov) | November 6, 2019 | 10/12/2019 | Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa) Patient Reported Outcomes in Adults With Pediatric-onset HypophosphatasiaTreated With Strensiq® (Asf ... | A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Tr ... | Hypophosphatasia | Drug: asfotase alfa | Alexion | Xcenda, LLC | Active, not recruiting | 18 Years | 99 Years | All | 100 | United States | |
| 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) Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypo ... | A Phase 2a, Randomized, Multicenter, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia A Phase 2a, Randomized, Multicenter, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotas ... | 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) Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset H ... | 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) An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (Human Recombinant Tissue-nons ... | 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 Asfotas ... | 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 Asfotas ... | 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] Hypophosphatasiais a rare inborn error of bone metabolism caused by inactivating mutations in the ge ... | 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 n ... | 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) An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (human recombinant tissue-nons ... | 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] Hypophosphatasia MedDRA version: 18.0;Level: PT;Classification code 10049933;Term: Hypophosphatasia; ... | 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 Trade Name: Strensiq Product Name: Asfotas Alfa Product Code: ENB-0040 INN or Proposed INN: ASFOTASE AL ... | 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 Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia(HPP) in J ... | 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 A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific ... | Hypophosphatasia | Biological: Asfotase Alfa | Alexion Pharmaceuticals | NULL | Completed | N/A | N/A | All | 13 | Phase 4 | NULL |