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Trial record 1 of 1 for:    NCT03790865
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Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome (ZEPHYR)

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ClinicalTrials.gov Identifier: NCT03790865
Recruitment Status : Recruiting
First Posted : January 1, 2019
Last Update Posted : June 12, 2019
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc. ( Millendo Therapeutics SAS )

Brief Summary:
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Hyperphagia Drug: Livoletide Drug: Placebo Phase 2 Phase 3

Detailed Description:

The protocol 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 followed by 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 may be initiated following review of safety and efficacy results at the completion of the Phase 2b Core Period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 26, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : March 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Low-Dose Livoletide
Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period.
Drug: Livoletide
Daily subcutaneous injection
Other Name: AZP-531

Experimental: High-Dose Livoletide
Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period.
Drug: Livoletide
Daily subcutaneous injection
Other Name: AZP-531

Placebo Comparator: Placebo
Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period.
Drug: Placebo
Daily subcutaneous injection




Primary Outcome Measures :
  1. Change in hyperphagia and food-related behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT) [ Time Frame: Baseline to month 3 ]
    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 :
  1. Change in fat mass [ Time Frame: Baseline to month 3 ]
    Percentage change from baseline to the end of the 3-month Core Period in total body fat mass in overweight/obese patients with PWS

  2. Change in waist circumference [ Time Frame: Baseline to month 3 ]
    Change from baseline to the end of the 3-month Core Period in waist circumference in overweight/obese patients with PWS

  3. Change in body weight [ Time Frame: Baseline to month 3 ]
    Percentage change from baseline to the end of the 3-month Core Period in body weight in overweight/obese patients with PWS



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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%

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03790865


Contacts
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Contact: Nadège Tremel +33 472 18 09 30 tremel@millendo.com
Contact: Robin Schmidt +1 734-845-9438 schmidt@millendo.com

Locations
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United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Hussein Abdul-Latif, MD         
United States, California
Rady Children's Hospital - San Diego Recruiting
San Diego, California, United States, 92123
Contact: Lynne Bird, MD         
United States, Colorado
Children's Hospital Colorado Not yet recruiting
Denver, Colorado, United States, 80045
Contact: Shawn McCandless, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Merlin Butler, MD         
United States, Michigan
University of Michigan Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Dan Shumer, MD         
United States, Minnesota
Children's Hospitals and Clinics of Minnesota-Minneapolis Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Jennifer Abuzzahab, MD         
United States, New York
Winthrop University Hospital Not yet recruiting
Mineola, New York, United States, 11501
Contact: Moris Angulo, MD         
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Contact: Italo Biaggioni, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Parisa Salehi, MD         
France
Centre Hospitalier Universitaire d'Angers Recruiting
Angers, France, 49100
Contact: Regis Coutant, MD         
Centre Hospitalier Universitaire d'Angers Recruiting
Angers, France, 49100
Contact: Frederic Illouz, MD         
CHU Lyon - Hopital Femmes Mere Enfant Recruiting
Bron, France, 69677
Contact: Marc Nicolino, MD         
Hospital Pitie Salpetriere Recruiting
Paris, France, 75013
Contact: Christine Poitou-Bernert, MD         
Hopital Necker-Enfants Malades Recruiting
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 Recruiting
Toulouse, France
Contact: Maithe Tauber, MD         
Spain
Hospital General Universitario de Alicante Recruiting
Alicante, Spain, 03010
Contact: Antonio Pico Alfonso, MI         
Hospital Sant Joan de Deu Recruiting
Barcelona, Spain, 08950
Contact: Marta Ramón, MD         
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell Recruiting
Sabadell, Spain, 08208
Contact: Assumpta Caixàs, MD         
Sponsors and Collaborators
Millendo Therapeutics SAS

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Responsible Party: Millendo Therapeutics SAS
ClinicalTrials.gov Identifier: NCT03790865     History of Changes
Other Study ID Numbers: AZP01-CLI-003
First Posted: January 1, 2019    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Millendo Therapeutics, Inc. ( Millendo Therapeutics SAS ):
Prader-Willi Syndrome
PWS

Additional relevant MeSH terms:
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Syndrome
Prader-Willi Syndrome
Hyperphagia
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Signs and Symptoms, Digestive
Signs and Symptoms