A Phase 2b/3 study to evaluate AZP-531 in PWS patients
Research type
Research Study
Full title
A Phase 2b/3 study to evaluate the safety, tolerability, and effects of Livoletide (AZP-531), an unacylated ghrelin analog, on food-related behaviors in patients with Prader-Willi syndrome.
IRAS ID
253405
Contact name
Anthony Goldstone
Contact email
Sponsor organisation
Millendo Therapeutics SAS
Eudract number
2018-003062-13
Duration of Study in the UK
1 years, 10 months, 25 days
Research summary
Prader-Willi syndrome (PWS) is a rare severe and life-threatening genetic disease characterised by hyperphagia (insatiable hunger) and abnormal eating behaviours.
Hyperphagia,and food related behaviors are associated with significant morbidity and represents a lifelong source of distress and severely and negatively affects social adaptation, occupational performance and quality of life.
This proposed study (funded by Millendo Therapeutics SAS) will evaluate the safety, tolerability, and effects of Livoletide on food related behaviours in patients with PWS.
This study includes 2 consecutive parts:
1. The first part is a Phase 2b dose-response study consisting of a 3 month double-blind, placebo-controlled Core Period and a 9-month Extension Period.
2. The second part is a Phase 3 study consisting of a 6-month double-blind, placebo-controlled Core Period followed by a 6 month Extension Period. Phase 3 will be initiated following review of safety and efficacy results at the completion of the Phase 2b Core Period.
For Phase 2b, one hundred and fifty (150) patients will be recruited and this part will last approximately 14 months. For Phase 3, the total number of required patients to be randomized is estimated to be 50 patients per group.
This study will be conducted at multiple centres worldwide, at approximately 25 centres in Europe,Australia and North America.
Participating sites will be specialised in the management of patients with PWS.
The primary objective of Phase 2b is to demonstrate the efficacy of a 3-month treatment with Livoletide as compared to placebo for reducing caregiver-observed food-related behaviour as assessed by Hyperphagia Questionnaire for Clinical Trials (HQ-CT).
REC name
London - Westminster Research Ethics Committee
REC reference
19/LO/0660
Date of REC Opinion
11 Jul 2019
REC opinion
Further Information Favourable Opinion