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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 : Terminated (Livoletide did not result in a statistically significant improvement in hyperphagia and food-related behaviors)
First Posted : January 2, 2019
Results First Posted : February 17, 2021
Last Update Posted : February 17, 2021
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.

A total of approximately 50 patients per group (8 to 65 years of age) will be randomized (approximately 150 patients in total). In addition to this cohort of 150 patients, a separate cohort of patients 4 to 7 years of age will also be randomized.

Note: The 8-65 year old cohort has been fully enrolled. Enrollment into the 4-7 year age cohort remains ongoing.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 158 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 Analogue, on Food-related Behaviors in Patients With Prader-Willi Syndrome
Actual Study Start Date : March 25, 2019
Actual Primary Completion Date : February 26, 2020
Actual Study Completion Date : May 25, 2020

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. Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]

    Total body fat mass was assessed using dual energy X-ray absorptiometry (DXA) scan. DXAs were conducted at each local facility using standardized procedures and settings.

    Overweight/obese patients were defined as follows:

    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

  2. Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]

    The waist circumference was measured in fasting condition at the superior border of iliac crest, according to recommendations from the Anthropometry Procedures Manual of the National Health and Nutrition Examination Survey, Revised 2007.

    Overweight/obese patients were defined as follows:

    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

  3. Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]

    Patients were weighed in fasting condition, clothed (underwear, light gown or light clothing), without footwear or heavy jewelry, using a calibrated scale. The same scale should be used throughout the study if possible. The conditions under which patients are weighed should be kept consistent if possible.

    Overweight/obese patients were defined as follows:

    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed genetic diagnosis of PWS
  • Evidence of increased appetite or hyperphagia
  • Patient must have a single primary caregiver who should be available for certain durations of the study
  • BMI ≤ 65 kg/m2
  • Growth hormone treatment permitted if doses have been stable for at least 1 month prior to screening

Exclusion Criteria:

  • History of chronic liver disease
  • Type 1 diabetes mellitus
  • HbA1c > 10%
  • Body weight <20 kg

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


Locations
Hide Hide 37 study locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, California
University of California - Irvine Medical Center
Orange, California, United States, 92868
Rady Children's Hospital - San Diego
San Diego, California, United States, 92123
United States, Colorado
Children's Hospital Colorado
Denver, Colorado, United States, 80045
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Children's Hospitals and Clinics of Minnesota-Minneapolis
Saint Paul, Minnesota, United States, 55102
United States, New York
Winthrop University Hospital
Mineola, New York, United States, 11501
New York Presbyterian Morgan Stanley Children's Hospital
New York, New York, United States, 10032
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Australia
Royal Prince Alfred Hospital
Camperdown, Australia, 2050
Austin Health
Melbourne, Australia, 3084
Perth Children's Hospital
Nedlands, Australia, 6009
The Childrens Hospital at Westmead
Westmead, Australia, 2145
Belgium
Cliniques Universitaires Saint-Luc
Brussels, Belgium, 1200
France
Centre Hospitalier Universitaire d'Angers
Angers, France, 49100
CHU Lyon - Hopital Femmes Mere Enfant
Bron, France, 69677
Hospital Pitie Salpetriere
Paris, France, 75013
Hopital Necker-Enfants Malades
Paris, France, 75015
CHU de Toulouse - Hospital Rangueil
Toulouse, France, 31059
CHU de Toulouse - Hopital des Enfants
Toulouse, France
Italy
Azienda Ospedaliera Universitaria Federico II
Napoli, Italy, 80131
Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Netherlands
Erasmus University Medical Center
Rotterdam, Netherlands, 3015
Stichting Kind en Groei
Rotterdam, Netherlands, 3016
Spain
Hospital General Universitario de Alicante
Alicante, Spain, 03010
Hospital de Cruces
Barakaldo, Spain, 48903
Hospital Sant Joan de Deu
Barcelona, Spain, 08950
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Sabadell, Spain, 08208
United Kingdom
NHS Tayside
Dundee, United Kingdom, DD1 9SY
Chelsea and Westminster Hospital
London, United Kingdom, SW109NH
Imperial College London
London, United Kingdom, W12 0NN
Sponsors and Collaborators
Millendo Therapeutics SAS
  Study Documents (Full-Text)

Documents provided by Millendo Therapeutics, Inc. ( Millendo Therapeutics SAS ):
Statistical Analysis Plan  [PDF] March 23, 2020
Study Protocol  [PDF] July 31, 2019

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Responsible Party: Millendo Therapeutics SAS
ClinicalTrials.gov Identifier: NCT03790865    
Other Study ID Numbers: AZP01-CLI-003
First Posted: January 2, 2019    Key Record Dates
Results First Posted: February 17, 2021
Last Update Posted: February 17, 2021
Last Verified: February 2021
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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Prader-Willi Syndrome
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