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Hypophosphatasia clinical trials

View clinical trials related to Hypophosphatasia.

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NCT ID: NCT02797821 Recruiting - Hypophosphatasia Clinical Trials

Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

Start date: June 2016
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of asfotase alfa in adult patients with pediatric-onset HPP.

NCT ID: NCT02603042 Recruiting - Hypophosphatasia Clinical Trials

Biomarker for Hypophosphatasia Disease

BioHypophos
Start date: November 2015
Phase: N/A
Study type: Observational

Development of a new MS-based biomarker for the early and sensitive diagnosis of Hypophosphatasia disease from plasma

NCT ID: NCT02531867 Completed - Hypophosphatasia Clinical Trials

Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan

Start date: July 2015
Phase: Phase 4
Study type: Interventional

This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.

NCT ID: NCT02496689 Approved for marketing - Hypophosphatasia Clinical Trials

Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

Start date: n/a
Phase: N/A
Study type: Expanded Access

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.

NCT ID: NCT02456038 Enrolling by invitation - Hypophosphatasia Clinical Trials

Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

Start date: August 2014
Phase: Phase 2
Study type: Interventional

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia

NCT ID: NCT02306720 Enrolling by invitation - Clinical trials for Hypophosphatasia (HPP)

An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Start date: December 2014
Phase: N/A
Study type: Observational [Patient Registry]

To collect information on the variability, progression, and natural history of HPP from patients of all ages, including infants, children, and adults with HPP, regardless of age at onset.

NCT ID: NCT02291497 Recruiting - Hypophosphatasia Clinical Trials

Burden of Disease in Hypophosphatasia (HPP)

Start date: October 2014
Phase: N/A
Study type: Observational

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

NCT ID: NCT02237625 Recruiting - Hypophosphatasia Clinical Trials

Natural History Study of Patients With Hypophosphatasia (HPP)

NatHisHPP
Start date: September 2014
Phase: N/A
Study type: Observational [Patient Registry]

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.

NCT ID: NCT02235493 Completed - Hypophosphatasia Clinical Trials

Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

Start date: August 2014
Phase: N/A
Study type: Observational

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.

NCT ID: NCT02104219 Completed - Clinical trials for Hypophosphatasia (HPP)

Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

Start date: March 2014
Phase: N/A
Study type: Observational

The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.