Hypophosphatasia Clinical Trial
Official title:
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)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.
U.S. sites participating in the expanded access program are closed to enrollment. ;
Status | Clinical Trial | Phase | |
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Completed |
NCT03418389 -
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
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Recruiting |
NCT02237625 -
Natural History Study of Patients With Hypophosphatasia (HPP)
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Completed |
NCT02291497 -
Burden of Disease in Hypophosphatasia (HPP)
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N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
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Active, not recruiting |
NCT04195763 -
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
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Not yet recruiting |
NCT05596539 -
Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
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Completed |
NCT02796885 -
Characterisation of Adult-Onset Hypophosphatasia
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Completed |
NCT02751801 -
Health Burden of Hypophosphatasia
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Completed |
NCT05890794 -
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
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Phase 1/Phase 2 | |
Recruiting |
NCT06079359 -
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
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Phase 3 | |
Recruiting |
NCT05234567 -
A Prospective Sub-Study of the Global Hypophosphatasia Registry
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Completed |
NCT02797821 -
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
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Phase 2 | |
Completed |
NCT01163149 -
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
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Phase 2 | |
Completed |
NCT04925804 -
Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
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Completed |
NCT02531867 -
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
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Phase 4 | |
Completed |
NCT01406977 -
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
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Phase 2 | |
Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Completed |
NCT01176266 -
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
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Phase 2/Phase 3 | |
Withdrawn |
NCT00894075 -
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
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Phase 2 | |
Active, not recruiting |
NCT04222452 -
The PORTRAIT Study
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