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.   

Recruitment Criteria

Study Type: not yet 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
Status:
 not yet recruiting
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 is anticipated to be available at the following sites:

United States, Alabama

University of Alabama at Birmingham                                                                 Not yet recruiting
Birmingham, Alabama, United States, 35233
Contact: Tushima Reeves    205-638-6452    treeves@peds.uab.edu  
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         mnrobins@uci.edu     
Principal Investigator: Virginia Kimonis, MD        

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

United States, Colorado

Children's Hospital Colorado                                                                                   Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Jacqueline Holstein    720-777-8439    jacqueline.holstein@childrenscolorado.org
Principal Investigator: Shawn McCandless, MD        

United States, Kansas

Kansas University Medical Center                                                                           Not yet recruiting
Kansas City, Kansas, United States, 66160
Contact: Yolanda Harness    913-588-1800    yharness@kumc.edu  
Principal Investigator: Merlin Butler, 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     andrea.hale@childrens.harvard.edu  
Principal Investigator: Amy Fleischman, MD      

United States, Maryland                                                                            

Johns Hopkins                                                                                                           Not yet recruiting
Baltimore, Maryland, United States, 21287
Contact: Kim Kafka   410-614-8583    kfehily2@jhmi.edu   
Principal Investigator: Ann Scheimann, MD  

United States, Michigan                                                                       

University of Michigan                                                                                              Not yet recruiting
Ann Arbor, Michigan, Unites States, 48109
Contact: Amy Hurst    734-615-1471     ahurst@med.umich.edu
Principal Investigator: Dan Shumer, MD        

United States, Minnesota

Children's Hospitals and Clinics of Minnesota                                                       Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus    651-220-5730    brittany.machus@childrensmn.org  
Principal Investigator: Jennifer Abuzzahab, MD        

United States, New York                                                                                    

New York Presbyterian Morgan Stanley Children's Hospital                                Not yet recruiting
New York, New York, United States, 10032
Contact: Joanne Carroll     212-305-1573     jc688@cumc.columbia.edu
Principal Investigator: Wendy Chung, MD                                                           

Winthrop University Hospital                                                                                   Not yet recruiting
Mineola, New York, United States, 11501
Contact: Marilyn Richardson     516-663-9582      marilyn.richardson@nyulangone.org
Principal Investigator: Moris Angulo, MD        

United States, Tennessee

Vanderbilt University School of Medicine                                                               Not yet recruiting
Nashville, Tennessee, United States, 37212
Contact: Elizabeth Roof    615-343-3330     elizabeth.roof@vanderbilt.edu
Principal Investigator: Italo Biaggioni MD         

United States, Texas                                                                                           

University of Texas Southwestern Medical Center                                                Not yet recruiting
Dallas, Texas, United States, 75390
Contact: Yasmin Dominguez      214-648-8718    yasmin.dominguez@childrens.com
Principal Investigator: Olga Gupta, MD        

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

United States, Washington

Seattle Children's Hospital                                                                                        Not yet recruiting
Seattle, Washington, United States, 98105         
Contact: Parisa Salehi     206-987-2640     parisa.salehi@seattlechildrens.org
Principal Investigator: Parisa Salehi, MD        

 

 

More information on Millendo and livoletide can be obtained at www.millendo.com

 

 

 

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