Clinical Trial: Propranolol for Prevention of Threshold Retinopathy of Prematurity

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Propranolol for Prevention of Threshold Retinopathy of Prematurity

Brief Summary: Extremely premature infants are at risk of developing a potentially blinding eye disease, called retinopathy of prematurity (ROP). Currently available treatment, consisting of laser surgery or injection of drugs into the eye balls, may prevent most but not all cases of permanent ROP-mediated blindness. Both types of treatment are associated with significant costs and side effects. An orally administered drug commonly used to treat hypertension, propranolol, may be effective in halting progression of ROP to severe stages, as suggested by preliminary data from two small studies. Propranolol has been used for decades not only in adult patients but also in newborn infants with heart diseases. Moreover, it has been licensed in 2014 for the use in newborn infants with hemangiomas in the European Union, Switzerland and the United States. As severe (threshold) ROP is an overall rare disease, the effectiveness of propranolol in combating ROP can only be assessed in a large, multicenter randomized controlled trial involving hospitals caring for extremely preterm infants of diverse origin.

Detailed Summary: Threshold Retinopathy of Prematurity (ROP), observed in a fraction of extremely premature infants, is characterized by retinal vessel proliferation that threatens vision secondary to retinal detachment. Currently available treatments (ablative laser surgery or intravitreal anti-VEGF injections) may prevent most but not all cases of permanent ROP-mediated blindness and are associated with significant costs and side effects. Orally administered propranolol, a commonly used drug to treat hypertension, may be effective in halting progression of ROP to severe stages, as suggested by preliminary data from two small studies. Propranolol has been used for decades not only in adult patients but also in newborn infants with heart diseases. Moreover, it has been licensed in 2014 for the use in newborn infants with hemangiomas in the European Union, Switzerland and the United States. This multicenter randomized placebo-controlled trial aims to assess whether oral propranolol given to extremely premature infants below 28 weeks gestational age reduces the rates of threshold ROP.
Sponsor: Charite University, Berlin, Germany

Current Primary Outcome: Survival without threshold ROP (stage 3 or more) [ Time Frame: 48 weeks p.m. gestational age ]

The primary endpoint for efficacy is survival until 48 weeks postmenstrual age without ROP stage 3 (any zone), as diagnosed according to the International Committee for the Classification of Retinopathy of Prematurity Revisited


Original Primary Outcome: Same as current

Current Secondary Outcome: Survival without ROP treated with ablative laser surgery or intravitreal antagonists of VEGF (vascular endothelial growth factor) [ Time Frame: 48 weeks p.m. gestational age ]

The secondary endpoint for efficacy is survival until 48 weeks postmenstrual age without local treatment for ROP (ablative laser surgery or intravitreal injections of anti-VEGF agents such as bevacizumab or ranibizumab)


Original Secondary Outcome: Same as current

Information By: Charite University, Berlin, Germany

Dates:
Date Received: March 13, 2017
Date Started: January 2018
Date Completion: August 2020
Last Updated: March 13, 2017
Last Verified: March 2017