PEDAL: Long-term Outcome of Children Enrolled in Study ROPP-2008-01

• Research type

  Research Study

• Full title

  Long-term Outcome of Children Enrolled in Study ROPP-2008-01 Previously Treated with rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity (ROP) or Who Received Standard Neonatal Care

• IRAS ID

  170560

• Contact name

  Mark Turner

• Contact email

  Mark.Turner@liverpool.ac.uk

• Sponsor organisation

  Premacure AB, A Member of the Shire Group of Companies

• Eudract number

  2014-003556-31

• Clinicaltrials.gov Identifier

  121698, IND number

• Duration of Study in the UK

  5 years, 0 months, 1 days

• Research summary

  Research Summary
  Retinopathy of prematurity (ROP) is a rare disorder of the developing retinal blood vessels and retinal neurons of the premature infant and is one of the leading causes of preventable blindness in children. Currently, there are no commercially available preventative treatments for ROP.

  Mecasermin rinfabate (rhIGF-1/rhIGFBP-3) is a drug approved for the treatment of growth failure in children.

  The ROPP-2008-01, Section D study was designed to compare the severity of retinopathy of prematurity (ROP) among treated infants with an untreated control population, while confirming the dose of rhIGF 1/rhIGFBP-3 is safe and efficacious.

  The purpose of this study is to continue to follow patients who participated in the ROPP-2008-01 study, to evaluate whether receiving rhIGF-1/rhIGFBP-3 for a short amount of time during that study has long term effects on vision or safety, as compared to the patients who received standard neonatal care during that study. This study will also help examine the long-term effects on cognitive, behavioural, and physical development, and Quality of Life.

  No study drug will be administered in this study.

  All 120 participants randomised in Section D of the ROPP-2008-01 study will be asked to participate in this long-term outcome study; approximately 40 patients are expected from the UK.

  The amount of time patients will participate in the study depends on their age when informed consent is obtained, however they will not be in the study beyond 5 ½ years of age. Study visits will take place at the age of 3 months (telephone), 6 months (site visit), 12 months (site visit), 20 months (site visit), 24 months (site visit), 3 years (telephone), 4 years (telephone), 4.75 years (site visit), 5 years (site visit).

  Procedures will include physical examinations, vital signs, electrocardiogram, visual assessments and behavioural assessments.

  This study is sponsored by Premacure AB, A Member of the Shire Group of Companies.

  Summary of Results
  Lay summary is still pending from sponsor

• REC name

  North East - York Research Ethics Committee

• REC reference

  15/NE/0052

• Date of REC Opinion

  26 Feb 2015

• REC opinion

  Further Information Favourable Opinion