Study of the the Effects of Tolvaptan in Children and Teens with ADPKD
Research type
Research Study
Full title
A Phase 3b, Two-part, Multicenter, One Year Randomized, Double-blind, Placebo-controlled Trial of the Safety, Pharmacokinetics, Tolerability, and Efficacy of Tolvaptan followed by a Two Year Open-label Extension in Children and Adolescent Subjects with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
IRAS ID
205250
Contact name
Detlef Bockenhauer
Contact email
Sponsor organisation
Otsuka Pharmaceutical Development & Commercialization, Inc.
Eudract number
2016-000187-42
Duration of Study in the UK
4 years, 1 months, 0 days
Research summary
Summary or Research
Tolvaptan is being studied to find out if tolvaptan is safe and helpful for children and teenagers with Autosomal Dominant Polycystic Kidney Disease (ADPKD). Children are being invited to take part in this research study because the study doctor has determined that they have ADPKD. This condition can arise as a result of a hereditary condition or a spontaneous genetic mutation. This condition affects how kidneys work over time. ADPKD can lead to infections, pain, high blood pressure, and kidney failure, although it may take many years before the children experiences these symptoms. Tests performed during the study will determine how much ADPKD will affected the children’s kidneys so far.
The purpose of this study is to look at the potential benefits and safety of tolvaptan in the paediatric and adolescent population with ADPKD. We will look at the number of cysts in the child’s kidneys, the child’s kidneys’ ability to function and the child’s ability to take the study drug without experiencing harmful side effects.
About 100 participants will be joining in this study globally or regionally at approximately 20 hospitals. There are two phases to this study: Phase A and Phase B. Phase A will last for 1 year and Phase B will last for an additional 2 years. If a child chooses to take part in Phase A of this study, and he/she completes it, they will have the option to enrol into Phase B.
Summary or Result
Would be made available by November 2022.
REC name
London - Central Research Ethics Committee
REC reference
16/LO/1012
Date of REC Opinion
4 Oct 2016
REC opinion
Further Information Favourable Opinion