Livoletide for the Treatment of PWS

Study Purpose

Livoletide (AZP-531) is a first-in-class analogue of unacylated ghrelin (UAG), a naturally occurring hormone that is thought to counteract the effects of acylated ghrelin (AG). ZEPHYR is a randomized, double-blind placebo-controlled study investigating Livoletide, which has been divided into two parts. The first part is a Phase 2b dose-response study consisting of a 3-month double-blind, placebo-controlled Core Period in which patients receive one of two doses of livoletide or placebo, followed by a 9-month Extension Period in which all patients will receive livoletide. The second part is a Phase 3 study consisting of a 6-month double-blind, placebo-controlled Core Period in which patients receive livoletide or placebo, followed by a 6-month Extension Period in which all patients will receive livoletide. The Phase 3 may be initiated following review of the safety and efficacy results at the completion of the Phase 2b Core Period.   

A webinar sharing details on this study was recorded April, 2019. You can watch the webinar below or read a transcription of the webinar on our blog

Recruitment Criteria

Status: recruiting
Eligible Ages: 8 - 65 years
Other Criteria: BMI < 65 kg/m2

Trial Details

ClinicalTrials.Gov IdentifierNCT03790865
Phase: 2b/3
Duration: Phase 2b will be 3 months followed by 9 month open label extension, Phase 3 will be 6 months double blind followed by 6-month open label extension
Lead Sponsor: Millendo Therapeutics SAS
Countries: United States, France, Spain, Italy, Belgium, Netherlands, Australia, United Kingdom
Additional Details: 

Eligibility Criteria

Inclusion Criteria:

  • Ages 8 to 65
  • Diagnosis of PWS confirmed by DNA methylation test
  • Evidence of increased appetite or hyperphagia
  • Patient must have a single primary caregiver who should be available for the duration of the study
  • BMI ≤ 65 kg/m2
  • Growth hormone treatment permitted if doses are stable

Exclusion Criteria:

  • History of chronic liver disease
  • Type 1 diabetes mellitus
  • HbA1c > 10%

Outcome Measures

This study will evaluate the drugs efficacy on the following:

Primary Outcome Measure:

  • Change in hyperphagia and food-related behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT): Change from baseline to the end of the 3-month Core Period for HQ-CT total score. The HQ-CT score range is 0 to 36 where the higher score represents more severe abnormal food-related behaviors.

Secondary Outcome Measures:

  • Change in fat mass: Percentage change from baseline to the end of the 3-month Core Period in total body fat mass in overweight/obese patients with PWS
  • Change in waist circumference: Change from baseline to the end of the 3-month Core Period in waist circumference in overweight/obese patients with PWS
  • Change in body weight: Percentage change from baseline to the end of the 3-month Core Period in body weight in overweight/obese patients with PWS

This study will be available at the following sites:

United States, Alabama

University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Contact: Tushima Reeves    205-638-6452  
Principal Investigator: Hussein Abdul-Latif, MD        

United States, California

University of California - Irvine Medical Center  - Not yet recruiting
Orange, California, United States, 92868
Contact: Margaret Knight     714-456-7004     
Principal Investigator: Virginia Kimonis, MD        

Rady Children's Hospital San Diego 
San Diego, California, United States, 92123
Contact: Michael Farrell    858-966-8450
Principal Investigator: Lynne Bird, MD        

United States, Colorado

Children's Hospital Colorado 
Aurora, Colorado, United States, 80045
Contact: Jacqueline Holstein    720-777-8439
Principal Investigator: Shawn McCandless, MD        

United States, Kansas

Kansas University Medical Center 
Kansas City, Kansas, United States, 66160
Contact: Yolanda Harness    913-588-1800  
Principal Investigator: Merlin Butler, MD        

United States, Maryland                                                                            

Johns Hopkins - Not yet recruiting
Baltimore, Maryland, United States, 21287
Contact: Kim Kafka   410-614-8583   
Principal Investigator: Ann Scheimann, MD 

United States, Massachusetts

University of Harvard Boston Children's Hospital - Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Andrea Hale       617-919-2867  
Principal Investigator: Amy Fleischman, MD       

United States, Michigan                                                                       

University of Michigan 
Ann Arbor, Michigan, Unites States, 48109
Contact: Amy Hurst    734-615-1471
Principal Investigator: Dan Shumer, MD        

United States, Minnesota

Children's Hospitals and Clinics of Minnesota 
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus    651-220-5730  
Principal Investigator: Jennifer Abuzzahab, MD        

United States, New York                                                                                    

New York Presbyterian Morgan Stanley Children's Hospital 
New York, New York, United States, 10032
Contact: Joanne Carroll     212-305-1573
Principal Investigator: Wendy Chung, MD                                                           

Winthrop University Hospital 
Mineola, New York, United States, 11501
Contact: Marilyn Richardson     516-663-9582
Principal Investigator: Moris Angulo, MD        

United States, Tennessee

Vanderbilt University School of Medicine 
Nashville, Tennessee, United States, 37212
Contact: Elizabeth Roof    615-343-3330
Principal Investigator: Italo Biaggioni MD         

United States, Texas                                                                                           

University of Texas Southwestern Medical Center 
Contact: Yasmin Dominguez      214-648-8718
Principal Investigator: Olga Gupta, MD        

Texas Children's Hospital - Not yet recruiting
Houston, Texas, United States, 77030
Contact: Debbie Barrera       832-824-4209  
Principal Investigator: Katherine Hwu, MD

United States, Washington

Seattle Children's Hospital 
Seattle, Washington, United States, 98105         
Contact: Parisa Salehi     206-987-2640
Principal Investigator: Parisa Salehi, MD        


Austin Health
Melbourne, Australia, 3084
Contact: Elif Ekinci, MD     


Cliniques Universitaires Saint-Luc 
Brussels, Belgium, 1200
Contact: Veronique Beauloye, PhD


Centre Hospitalier Universitaire d'Angers 
Angers, France, 49100
Contact: Regis Coutant, MD         

Centre Hospitalier Universitaire d'Angers
Angers, France, 49100
Contact: Frederic Illouz, MD         

CHU Lyon - Hopital Femmes Mere Enfant 
Bron, France, 69677
Contact: Marc Nicolino, MD         

Hospital Pitie Salpetriere 
Paris, France, 75013
Contact: Christine Poitou-Bernert, MD   

Hopital Necker-Enfants Malades
Paris, France, 75015
Contact: Graziella Pinto, MD         

CHU de Toulouse - Hospital Rangueil - Not yet recruiting
Toulouse, France, 31059
Contact: Patrick Ritz, MD          

CHU de Toulouse - Hopital des Enfants 
Toulouse, France
Contact: Maithe Tauber, MD          


Azienda Ospedaliera Universitaria Federico II                                   
Napoli, Italy, 80131
Contact: Graziano Grugni, MD         


Erasmus University Medical Center - Not yet recruiting
Rotterdam, Netherlands, 3015
Contact: Laura De Graaff-Herder, MD 


Hospital General Universitario de Alicante 
Alicante, Spain, 03010
Contact: Antonio Pico Alfonso, MI          

Hospital Sant Joan de Deu 
Barcelona, Spain, 08950
Contact: Marta Ramón, MD         

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell 
Sabadell, Spain, 08208
Contact: Assumpta Caixàs, MD   

United Kingdom

NHS Tayside - Not yet recruiting
Dundee, United Kingdom, DD1 9SY
Contact: Nicholas Conway, MD    

More information on Millendo and livoletide can be obtained at




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