Clinical trials: Hypophosphatasia (HPP)
Hypophosphatasia
Natural History Study of Patients With Hypophosphatasia (HPP)
Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by defective bone and teeth mineralization caused by mutations of the ALPL gene, which encodes for the tissue-nonspecific alkaline phosphatase (TNSALP)…
- Trial name
- NatHisHPP (NCT02237625)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Duke University
Hypophosphatasia
Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)
The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia
- Trial name
- NCT02456038
- Testing
- Asfotase Alfa (ALXN1215)
- Where
- 12 sites in Japan
- Volunteers
- 13 people
- Sponsor
- Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
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 (HPP)
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia…
- Trial name
- NCT07179640
- Testing
- ALE1
- Where
- 3 sites in Germany, New Zealand and 1 more
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Alesta Therapeutics
Hypophosphatasia
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- MULBERRY (NCT06079359)
- Testing
- ALXN1850
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 30 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
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 (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 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)
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
Burden of Disease in Hypophosphatasia (HPP)
Hypophosphatasia (HPP) is a rare, inherited metabolic disease caused by inactivating mutations in the Alkaline Phosphatase (ALPL) gene, coding for the Tissue-nonspecific alkaline phosphatase (TNAP).
- Trial name
- NCT02291497
- Where
- Germany
- Volunteers
- 114 people
- Sponsor
- Wuerzburg University Hospital
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
The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS)
- Trial name
- ATLANTIS (NCT07390240)
- Where
- 4 sites in Russia
- Volunteers
- 55 people
- Sponsor
- AstraZeneca
Hypophosphatasia (HPP)
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
- Trial name
- NCT01203826
- Testing
- Asfotase Alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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 (HPP)
Registry of Patients With Hypophosphatasia
In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.
- Trial name
- NCT02306720
- Where
- 66 sites in United States, Australia and 10 more
- Volunteers
- 1,571 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.
Osteoporosis
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
The study is designed as a retrospective, multicenter study for subjects previously treated with the AGN1 LOEP Kit in the proximal femur as part of standard hospital practice. This will be a non-randomized and non-blinded study.
- Trial name
- NCT07594639
- Testing
- AGN1 LOEP
- Where
- Belgium
- Volunteers
- 305 people
- Sponsor
- AgNovos Healthcare, LLC
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
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- HICKORY (NCT06079281)
- Testing
- ALXN1850
- Where
- 65 sites in United States, Argentina and 15 more
- Volunteers
- 124 people
- Sponsor
- Alexion Pharmaceuticals, Inc.