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Hypophosphatasia Clinical Trials

Browse current & upcoming clinical research / studies on Hypophosphatasia. There are a total of 8 clinical trials for Hypophosphatasia in 12 countries with 4 trials currently in the United States. 4 are either active and/or recruiting patients or have not yet been completed. Click the title of each study to get the complete details on eligibility, location & other facts about the study.

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Definitions
Interventional trials
Determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments.
Observational trials
Address health issues in large groups of people or populations in natural settings.
Recruiting
Participants are currently being recruited and enrolled.
Active, not recruiting
Study is ongoing (i.e., patients are being treated or examined), but enrollment has completed.
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Participants are not yet being recruited or enrolled.
Enrolling by invitation
Participants are being (or will be) selected from a predetermined population.
Completed
The study has concluded normally; participants are no longer being examined or treated (i.e., last patient's last visit has occurred).
Withdrawn
Study halted prematurely, prior to enrollment of first participant.
Suspended
Recruiting or enrolling participants has halted prematurely but potentially will resume.
Terminated
Recruiting or enrolling participants has halted prematurely and will not resume; participants are no longer being examined or treated.
October 2014 -
Hypophosphatasia (HPP) is a rare, inherited metabolic disease caused by inactivating mutations in the ALPL gene, coding for the Tissue-nonspecific alkaline phosphatase (TNAP). Penetrance and disease severity is very heterogenous, ranging from stillbirth to adult-onset manifestations. Especially the latter are again characterized by an extremely broad spectrum of symptoms. This scope of variability makes it difficult to attribute individual patients' symptoms to the disease and distinguish them from HPP independent health issues. Especially in adult HPP patients, musculoskeletal problems, including (fragility-) fractures / bone bruise, joint pain, reduced mobility, muscular weakness and pain and reduced muscular endurance appear to reflect the prevailing burden of disease, especially with respect these patients dis-abilities of daily life. To expand current knowledge of the natural history of the disease as well as on disease specific musculoskeletal deficits in HPP, all adult patients with established Diagnosis of HPP known at the Orthopedic Insitute, University of Würzburg, will be offered to participate in a single, multimodal assessment of their disease history, current symptoms and disabilities, lab evaluations and clinical and technical analysis of their musculoskeletal status and capabilities. Patients will be invited to a day long visit to the clinic in order to perform the following assessments: A) Epidemiologic / anamnestic information B) Physical examination C) Structured questionnaires D) Laboratory examinations E) Clinical functional testing F) Technical Examinations
Sponsor: Wuerzburg University Hospital
Study type: Observational
September 2014 - September 2026
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) isozyme, resulting in decreased serum and bone alkaline phosphatase levels. To date, over 250 different mutations in the gene encoding TNSALP have been associated with HPP. Clinically, the loss of TNSALP function results in progressive skeletal impact as well as progressive impact on all other major organ systems. It clinically manifests as rickets in infants and children and osteomalacia at all ages. The severe form of the disease has been estimated to have a prevalence of about 1 in every 100,000 live births.
Sponsor: Duke University
Study type: Observational [P
July 2011 - September 2012
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. This study will allow a comparison of several doses of the study drug within the first two weeks after administration and after a longer assessment period for the highest dose level to enable selection of dose ranges to be tested in subsequent studies in the HPP indication.
Sponsor: Novartis
Study type: Interventional
July 2010 - December 2015
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. The purpose of this study is to test the safety and efficacy of a study drug called asfotase alfa (human recombinant tissue non-specific alkaline phosphate fusion protein) to see what effects it has on patients = 5 years of age or less with HPP.
Sponsor: Alexion Pharma International Sarl
Study type: Interventional
June 2010 - December 2015
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. The purpose of this study is to test the safety and efficacy of two doses of the study drug called asfotase alfa as compared to a control group to see what effects it has adolescents and adults with HPP.
Sponsor: Alexion Pharma International Sarl
Study type: Interventional
July 2009 - December 2014
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. The purpose of this study is to test the safety of the study drug called ENB-0040 and see what effects is has on human juveniles and HPP.
Sponsor: Alexion Pharma International Sarl
Study type: Interventional
April 2009 - December 2015
This clinical trial studies the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed the ENB-002-08 study. Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).
Sponsor: Alexion Pharma International Sarl
Study type: Interventional
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The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
Sponsor: Alexion Pharma International Sarl
Study type: Observational
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