Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

— ZEPHYR  

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03790865
• Other study ID #: AZP01-CLI-003
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: March 25, 2019
• Est. completion date: May 25, 2020

Study information

• Verified date: February 2021
• Source: Millendo Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 158
• Est. completion date: May 25, 2020
• Est. primary completion date: February 26, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Hyperphagia
• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Livoletide
Daily subcutaneous injection
• Placebo
Daily subcutaneous injection

Locations

Country	Name	City	State
Australia	Royal Prince Alfred Hospital	Camperdown
Australia	Austin Health	Melbourne
Australia	Perth Children's Hospital	Nedlands
Australia	The Childrens Hospital at Westmead	Westmead
Belgium	Cliniques Universitaires Saint-Luc	Brussels
France	Centre Hospitalier Universitaire d'Angers	Angers
France	CHU Lyon - Hopital Femmes Mere Enfant	Bron
France	Hopital Necker-Enfants Malades	Paris
France	Hospital Pitie Salpetriere	Paris
France	CHU de Toulouse - Hopital des Enfants	Toulouse
France	CHU de Toulouse - Hospital Rangueil	Toulouse
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli
Italy	Ospedale Pediatrico Bambino Gesù	Roma
Netherlands	Erasmus University Medical Center	Rotterdam
Netherlands	Stichting Kind en Groei	Rotterdam
Spain	Hospital General Universitario de Alicante	Alicante
Spain	Hospital de Cruces	Barakaldo
Spain	Hospital Sant Joan de Deu	Barcelona
Spain	Corporacio Sanitaria Parc Tauli - Hospital de Sabadell	Sabadell
United Kingdom	NHS Tayside	Dundee
United Kingdom	Chelsea and Westminster Hospital	London
United Kingdom	Imperial College London	London
United States	University of Michigan	Ann Arbor	Michigan
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	Children's Hospital Colorado	Denver	Colorado
United States	Baylor College of Medicine	Houston	Texas
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Winthrop University Hospital	Mineola	New York
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	New York Presbyterian Morgan Stanley Children's Hospital	New York	New York
United States	University of California - Irvine Medical Center	Orange	California
United States	Children's Hospitals and Clinics of Minnesota-Minneapolis	Saint Paul	Minnesota
United States	Rady Children's Hospital - San Diego	San Diego	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Millendo Therapeutics SAS

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  France,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline to month 3
Secondary	Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese	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	Baseline to month 3
Secondary	Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese	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	Baseline to month 3
Secondary	Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese	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	Baseline to month 3