DMID 11-0069 Valgan Toddler, Version 2.0 Dated 30 May 2014
Research type
Research Study
Full title
A PHASE II RANDOMIZED AND CONTROLLED INVESTIGATION OF SIX WEEKS OF ORAL VALGANCICLOVIR THERAPY IN INFANTS AND CHILDREN WITH CONGENITAL CYTOMEGALOVIRUS INFECTION AND HEARING LOSS.
IRAS ID
156798
Contact name
Paul David Griffiths
Contact email
Sponsor organisation
University College London
Eudract number
2014-001920-31
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
08-0172, The UK Sponsor is UCL
Duration of Study in the UK
4 years, 1 months, 28 days
Research summary
Congenital cytomegalovirus affects 7/1000 babies. Approximately 13% are born with symptoms, half of whom develop neurological impairment or sensorineural hearing-loss. Of the remainder, approximately 12% develop symptoms on follow-up, making congenital CMV infection a common cause of sensorineural hearing loss.
A controlled trial in the USA recruited neonates born with symptoms and randomised them to receive 6 weeks of intravenous ganciclovir or no treatment. The results showed significantly worse hearing at 6 months of age among those who were not treated and established ganciclovir as the standard of care for neonates. This group then used oral valganciclovir to produce blood levels of ganciclovir equivalent to those in the original trial. This dose of valganciclovir was then used in a double-blind, controlled trial to treat neonates born with symptoms for 6 weeks and then randomise them to complete a 6 month course with valganciclovir or matching placebo. To help with recruitment and share expertise, centres in the UK (chief investigator Prof Paul Griffiths) joined in this study.
Children who present with sensorineural hearing loss can now have their dried blood spot taken as a neonate retrieved and tested for CMV DNA to diagnose congenital CMV infection. The stage is now set to address important unmet medical need by combining this new diagnostic method with a new randomised controlled trial.
We will recruit children aged one month up to their 4th birthday who have sensorineural hearing-loss and congenital CMV infection. We will offer their parents recruitment into a double-blind randomised placebo-controlled study of valganciclovir given for 6 weeks. The primary endpoint will be sensorineural hearing-loss and the study has been powered to replicate the difference seen in the original randomised controlled trial of ganciclovir in neonates.
This study has the potential to identify a new treatment for a currently untreatable important disease.REC name
London - City & East Research Ethics Committee
REC reference
14/LO/1578
Date of REC Opinion
9 Dec 2014
REC opinion
Further Information Favourable Opinion