Clinical trials: Hypophosphatasia
Hypophosphatasia
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support…
- Trial name
- NCT04195763
- Testing
- asfotase alfa
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Characteristics of Hypophosphatasia in Adult Patients in Rheumatology
With hypophosphatasia still being frequently overlooked and misdiagnosed, the primary aim of this prospective observational study is to determine the prevalence of hypophosphatasia in adult patients in rheumatology, and beyond…
- Trial name
- COHIR (NCT06161142)
- Testing
- Second alkaline phosphatase measurement, Extended laboratory diagnostics, Symptom and clinical findings checklist for hypophosphatasia
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- University of Bonn
Hypophosphatasia
Characterisation of Adult-Onset Hypophosphatasia
Hypophosphatasia (HPP) is an inherited condition which causes a defect in bone calcification, leading to weak bones. Early childhood forms are severe and easily recognised, and there is now a drug treatment which is very…
- Trial name
- NCT02796885
- Testing
- no intervention - observational study
- Where
- 2 sites in United Kingdom
- Volunteers
- 60 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Hypophosphatasia
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT00894075
- Testing
- ENB-0040
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia, Bone Diseases, Metabolic
Circulating miRNAs and Bone Microstructure in Adults With Hypophosphatasia
The aim of the study is to accomplish a complete bone status of patients with HPP using new approaches to assess bone quality.
- Conditions
- Hypophosphatasia, Bone Diseases, Metabolic, Bone
- Trial name
- NCT04018287
- Testing
- HR-pQCT scans, BMD measurements, bone specific circulating microRNAs (miRNAs)
- Where
- Austria
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Medical University of Vienna
Hypophosphatasia
The PORTRAIT Study
Clinical Consequences of Adults Presenting with Hypophosphatasia with Special Focus on Gait, Bone Microstructure and Cognition: The PORTRAIT study Hypophosphatasia (HPP) is an inherited condition that leads to weak bones.
- Trial name
- PORTRAIT (NCT04222452)
- Where
- United Kingdom
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Hypophosphatasia
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
The purpose of the study is to determine tolerability, PK/PD and preliminary efficacy of BPS804 in adult patients with HPP treated with multiple escalating doses of BPS804.
- Trial name
- NCT01406977
- Testing
- BPS804
- Where
- Germany
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hypophosphatasia
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and symptoms, most commonly…
- Trial name
- EmPATHY (NCT03418389)
- Where
- Germany
- Volunteers
- 23 people
- Sponsor
- Dr. Lothar Seefried
Hypophosphatasia
Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
Observational study to perform Whole Genome Sequencing in participants clinically suspected for HPP and negative for known pathogenic ALPL variants
- Trial name
- NCT04925804
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- CENTOGENE GmbH Rostock
Hypophosphatasia (HPP)
Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP).
- Trial name
- BABS (NCT04181164)
- Testing
- Microindentation, High resolution peripheral quantitative computed tomography (HRpQCT), Biochemical analysis of different bone markers.
- Where
- Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hvidovre University Hospital
Hypophosphatasia
Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
- Trial name
- REBUILD (NCT04189315)
- Testing
- asfotase alfa
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
The purpose of this study was to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of asfotase alfa in adult participants with pediatric-onset HPP.
- Trial name
- NCT02797821
- Testing
- Asfotase alfa
- Where
- 4 sites in United States, Germany
- Volunteers
- 27 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
- Trial name
- NCT00744042
- Testing
- asfotase alfa
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
- Trial name
- NCT00952484
- Testing
- asfotase alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.
- Trial name
- NCT02104219
- Where
- 19 sites in United States, Australia and 6 more
- Volunteers
- 32 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
- Trial name
- NCT00739505
- Testing
- Asfotase Alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Expanded Access Program for Asfotase Alfa 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.
- Trial name
- NCT02496689
- Testing
- asfotase alfa
- Where
- 5 sites in United States, France
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01163149
- Testing
- asfotase alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 19 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01176266
- Testing
- asfotase alfa
- Where
- 23 sites in United States, Australia and 10 more
- Volunteers
- 69 people
- Sponsor
- Alexion Pharmaceuticals, Inc.