Compassionate use of ENB-0040 for infantile hypophosphatasia

• Research type

  Research Study

• Full title

  A Multicenter, Open Label Study of the Safety, Tolerability and Pharmacology of ENB-0040 (Enobia's human recombinant tissue non-specific alkaline phosphatase fusion protein) in up to 10 Severely Affected Patients with Infantile Hypophosphatasia (HPP)

• IRAS ID

  12586

• Contact name

  Nick Bishop

• Sponsor organisation

  Enobia Canada Limited Partnership

• Eudract number

  2008-007406-11

• Research summary

  Two sites in the UK will assess the study drug when given to patients aged 36 months or under with severe infantile 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. The study will assess the safety of the treatment and look at changes in the patient??s HPP symptoms, growth, development and specifically bone formation.Eligible infants will receive a single injection of the study drug into a vein (intravenously) followed by subcutaneous injections (just under the skin) three times a week for 6 months. Intensive monitoring of the patients will be performed following treatments and assessments will include review of child development, physical examinations, eye examinations, x-rays, measurements of vital signs (pulse rate, blood pressure and temperature) and child'size. Blood and urine will be sampled to assess safety, drug levels and bone and disease markers. Patients who complete the study will be eligible for inclusion in a long-term extension study.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  09/H1008/15

• Date of REC Opinion

  11 Feb 2009

• REC opinion

  Favourable Opinion