Extension Study of ENB-0040 in Severely Affected Infants and Young Chil

• Research type

  Research Study

• Full title

  Extension Study of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children with Hypophosphatasia (HPP).

• IRAS ID

  15736

• Eudract number

  2009-009369-32

• Research summary

  This is a 1-year extension to the ENB-002-08 study: a study of an experimental drug (ENB-0040) in as many as 6 infants with severe hypophosphatasia (HPP). HPP is a rare bone disorder caused by gene mutations or changes which result in low levels of an enzyme needed to harden and develop bones. Two sites in the UK will continue to assess the study drug ENB-0040 when given to eligible patients, aged 42 months or under, that have completed the ENB-002-08 study. This study will assess the long-term safety and tolerability of the treatment and look at changes in the patient??s HPP symptoms, growth, development and specifically bone formation.Eligible infants will continue the same dose of subcutaneous injections (just under the skin) of ENB-0040 as established in study ENB-002-08, with provision for dose adjustment, three times a week for one year. Study visits will occur approximately every three months and will include the following assessments: review of child development and growth, physical examinations, eye examinations, x-rays, measurements of vital signs (pulse rate, blood pressure, breathing rate and temperature), and blood and urine samples (to assess safety, drug levels and bone and disease markers).

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  09/H1008/40

• Date of REC Opinion

  30 Mar 2009

• REC opinion

  Further Information Favourable Opinion