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Safety and Efficacy Study of ENB-0040 in Adolescents and Adults With Hypophosphatasia (HPP)

2014-08-27 03:12:11 | BioPortfolio

Summary

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 ENB-0040 as compared to a control group to see what effects it has adolescents and adults with HPP.

Description

Hypophosphatasia is rare inherited form of rickets and osteomalacia caused by inactivating mutations in the gene encoding the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). The prevalence of the disease is thought to be about 1:100,000. Inheritance can be autosomal recessive or dominant, and penetrance is variable resulting in a wide range of clinical expressivity. HPP differs from other forms of rickets and osteomalacia in that serum levels of calcium and phosphorus are generally normal or even elevated. Low circulating levels of alkaline phosphatase with elevated serum or urine levels of the TNSALP substrates, inorganic pyrophosphate (PPi), pyroxidal 5'-phosphate (PLP) and phosphoethanolamine (PEA), are the biochemical hallmarks of this inborn error of metabolism.

Disease severity is inversely related to the age at symptom presentation. The most severe cases occur in utero and almost invariably result in death, generally due to pulmonary compromise. Infants who present in the first six months of life have a mortality rate of approximately 50%. Children and adults have less severe disease but can have frequent fractures, bone pain, bowing of the long bones and muscle weakness. Morbidity is generally cumulative; some patients cannot ambulate independently and end up wheelchair-bound.

Study Design

Allocation: Randomized, Control: Dose Comparison, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Hypophosphatasia

Intervention

ENB-0040, ENB-0040

Location

Duke University Medical Center
Durham
North Carolina
United States
27710

Status

Recruiting

Source

Enobia Pharma

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:12:11-0400

Clinical Trials [9 Associated Clinical Trials listed on BioPortfolio]

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein (ENB-0040) in Adults 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 ha...

Safety Study of ENB-0040 in Severely Affected Infants 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 h...

16.0040 Ankylosing Spondylitis Study

The purpose of this study was to evaluate extended safety and efficacy of etanercept in adults with Ankylosing Spondylitis.

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 ...

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

This clinical trial is being conducted in Hypophosphatasia (HPP), a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme n...

PubMed Articles [6 Associated PubMed Articles listed on BioPortfolio]

Ecotoxicological effects of the insecticide fipronil in Brazilian native stingless bees Melipona scutellaris (Apidae: Meliponini).

Melipona scutellaris Latreille, 1811 (Hymenoptera, Apidae) is a pollinator of various native and cultivated plants. Because of the expansion of agriculture and the need to ensure pest control, the use...

Rare variants in the gene ALPL that cause hypophosphatasia are strongly associated with ovarian and uterine disorders.

Mutations in ALPL, which encodes tissue-nonspecific isozyme alkaline phosphatase (TNSALP), cause hypophosphatasia (HPP). HPP is suspected by a low serum alkaline phosphatase (AlkP). We hypothesized th...

Highly efficient infrared to visible up-conversion emission tuning from red to white in Eu/Yb co-doped NaYF phosphor.

Eu/Yb co-doped NaYF phosphors have been synthesized by the combustion method. The Eu doping was fixed and the effect of Yb doping concentration on the structural, morphological and luminescence proper...

Evaluation of silicon membranes for extracorporeal membrane oxygenation (ECMO).

While extracorporeal membrane oxygenation (ECMO) is a valuable therapy for patients with lung or heart failure, clinical use of ECMO remains limited due to hemocompatibility concerns with pro-coagulat...

Spinal muscular atrophy within Amish and Mennonite populations: Ancestral haplotypes and natural history.

We correlate chromosome 5 haplotypes and SMN2 copy number with disease expression in 42 Mennonite and 14 Amish patients with spinal muscular atrophy (SMA). A single haplotype (A1) with 1 copy of SMN2 ...

Medical and Biotech [MESH] Definitions

An inorganic pyrophosphate which affects calcium metabolism in mammals. Abnormalities in its metabolism occur in some human diseases, notably HYPOPHOSPHATASIA and pseudogout (CHONDROCALCINOSIS).

A genetic metabolic disorder resulting from serum and bone alkaline phosphatase deficiency leading to hypercalcemia, ethanolamine phosphatemia, and ethanolamine phosphaturia. Clinical manifestations include severe skeletal defects resembling vitamin D-resistant rickets, failure of the calvarium to calcify, dyspnea, cyanosis, vomiting, constipation, renal calcinosis, failure to thrive, disorders of movement, beading of the costochondral junction, and rachitic bone changes. (From Dorland, 27th ed)

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