WN39434-Phase III study of Balovaptan in Adults with ASD
Research type
Research Study
Full title
A PHASE III, RANDOMIZED, DOUBLE-BLIND,PLACEBO-CONTROLLED, EFFICACY, AND SAFETY STUDY OF BALOVAPTAN IN ADULTS WITH AUTISM SPECTRUM DISORDER WITH A 2-YEAR OPEN-LABEL EXTENSION
IRAS ID
246929
Contact name
Declan Murphy
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2017-004378-32
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
4 years, 1 months, 3 days
Research summary
Currently there is no specific treatment to manage the social and communication symptoms for people living with autism spectrum disorder (ASD). Consequently, there is a high unmet need for effective therapies to treat the main symptoms in ASD.
The purpose of this research study is to compare the effects, good or bad, of a new medicine called balovaptan with a placebo (an inactive substance that looks like balovaptan) on social behaviour and communication in adults with ASD. Balovaptan is an experimental medicine and has not been approved by the health authorities for the treatment of ASD.
The study will enrol approximately 350 participants globally who will be randomly allocated to one of two treatment arms:
Arm A: Balovaptan
Arm B: Placebo.
Neither the participant or doctor will know which treatment has been allocated, this is known as a double blind study. There is a 50% chance to receive balovaptan during the double blind treatment.Participants that have a confirmed diagnosis of ASD and a family member or friend to support them with study visits maybe eligible to participate in the study.
The study will consist of the following stages:
• Screening (approx. 1 month)
• Double Blind Treatment (approx. 6 months)
• Open-Label Treatment Extension (approx. 2 years)
• Follow-Up Period (approx.3 months)
Participants that complete the 6 months of double blind treatment, have the option to participate in the open label extension treatment and have a 100% chance of receiving the study drug balovaptan.The total length of the study is approximately 4-5 years, which is based on the recruitment period taking between 18-28 months.
Approximately 16 participants will be recruited at 4 UK hospitals.
The study is sponsored by F. Hoffman La Roche Ltd
Research Summary; Version Number 1.0, dated: 03rd July 2018
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0248
Date of REC Opinion
18 Sep 2018
REC opinion
Further Information Favourable Opinion