RAINBOW extension study
Research type
Research Study
Full title
RAINBOW extension study: an extension study to evaluate the long term efficacy and safety of ranibizumab compared with laser therapy for the treatment of infants born prematurely with retinopathy of prematurity
IRAS ID
202764
Contact name
Eva-Maria Simank
Contact email
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2014-004048-36
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
000527-PIP04-13, PIP number
Duration of Study in the UK
5 years, 3 months, 6 days
Research summary
Summary of Research
During foetal development, the retina develops late in the womb and very premature babies may have incomplete development of the blood vessels needed to provide oxygen to the retina. After birth, blood vessels may develop abnormally causing vision loss/blindness. Retinopathy of prematurity (ROP) affects ~20% of premature babies, is a significant cause of blindness in children in developed and developing countries with ~50,000 children blind from ROP worldwide.Laser therapy to the eye is the standard treatment for ROP with earlier treatment shown to be more effective, however this is not possible for some patients and usually requires general anaesthesia and good accuracy of visualizing the eye, both difficult to achieve with premature babies.
New blood vessel formation requires a protein called vascular endothelial growth factor (VEGF). Ranibizumab is a drug which is injected into the eye and works by blocking the effects of VEGF to prevent the growth of these abnormal blood vessels.
This is an extension study for the core 24 week open-label study (the study doctor, parent(s)/legal guardian(s) will know which treatment is being received) to compare 2 different doses of ranibizumab (0.1mg and 0.2mg) with standard laser therapy. The purpose of this extension study is to evaluate the long-term efficacy and safety of ranibizumab compared with laser therapy in infants enrolled in the core study up to their 5th birthday and treatment with ranibizumab is permitted for eligible eyes up to and including week 40 from the baseline visit in the core study. The remainder of the extension study is observational.
Premature babies with ROP who have successfully completed the 24 week core study will be included in this study with a target of 12 patients in the UK across 6 sites.
Summary of results
: Preterm infants with ROP treated with ranibizumab 0.2 mg showed better visual acuity, higher rate of absence of ocular structural abnormalities, and reduced rates of myopia and high myopia compared with laser therapy at the patients’ 5th birthday visit. • Ranibizumab 0.2 mg is safe and well-tolerated in patients with ROP up to the patients’ 5th birthday. The long-term safety profile of ranibizumab 0.2 mg was in line with safety data from the core study, with no new safety signals observed.REC name
London - London Bridge Research Ethics Committee
REC reference
16/LO/0794
Date of REC Opinion
5 Jul 2016
REC opinion
Favourable Opinion