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

NCT03790865 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: AZP01-CLI-003
• Study Type: interventional
• Target: 158
• Locations: 8 countries
• Sites: 37

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).

Conditions

Prader-Willi Syndrome; Hyperphagia

Keywords

Prader-Willi Syndrome; PWS

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese

  Baseline to month 3

• Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese

  Baseline to month 3

• Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese

  Baseline to month 3

Study Arms (3)

Low-Dose Livoletide

EXPERIMENTAL

Daily subcutaneous injection of \~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Drug: Livoletide

High-Dose Livoletide

EXPERIMENTAL

Daily subcutaneous injection of \~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Drug: Livoletide

Placebo

PLACEBO COMPARATOR

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

Interventions

Livoletide DRUG

Daily subcutaneous injection

Also known as: AZP-531

High-Dose Livoletide; Low-Dose Livoletide

Placebo DRUG

Daily subcutaneous injection

Placebo

Eligibility Criteria

• Age: 4 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Millendo Therapeutics SAS: lead

Study Sites (37)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

University of California - Irvine Medical Center

Orange, California, 92868, United States

Location

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Denver, Colorado, 80045, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospitals and Clinics of Minnesota-Minneapolis

Saint Paul, Minnesota, 55102, United States

Location

Winthrop University Hospital

Mineola, New York, 11501, United States

Location

New York Presbyterian Morgan Stanley Children's Hospital

New York, New York, 10032, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

Location

Austin Health

Melbourne, 3084, Australia

Location

Perth Children's Hospital

Nedlands, 6009, Australia

Location

The Childrens Hospital at Westmead

Westmead, 2145, Australia

Location

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

Location

Centre Hospitalier Universitaire d'Angers

Angers, 49100, France

Location

CHU Lyon - Hopital Femmes Mere Enfant

Bron, 69677, France

Location

Hospital Pitie Salpetriere

Paris, 75013, France

Location

Hopital Necker-Enfants Malades

Paris, 75015, France

Location

CHU de Toulouse - Hospital Rangueil

Toulouse, 31059, France

Location

CHU de Toulouse - Hopital des Enfants

Toulouse, France

Location

Azienda Ospedaliera Universitaria Federico II

Napoli, 80131, Italy

Location

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

Erasmus University Medical Center

Rotterdam, 3015, Netherlands

Location

Stichting Kind en Groei

Rotterdam, 3016, Netherlands

Location

Hospital General Universitario de Alicante

Alicante, 03010, Spain

Location

Hospital de Cruces

Barakaldo, 48903, Spain

Location

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Sabadell, 08208, Spain

Location

NHS Tayside

Dundee, DD1 9SY, United Kingdom

Location

Chelsea and Westminster Hospital

London, SW109NH, United Kingdom

Location

Imperial College London

London, W12 0NN, United Kingdom

Location

MeSH Terms

Conditions

Prader-Willi Syndrome; Hyperphagia

Interventions

cyclic des-acyl ghrelin (6-13)

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

The double-blind, placebo-controlled phase 2b study did not meet the primary endpoint or any of the secondary endpoints. Therefore, the Sponsor has decided to discontinue further development of livoletide.

Results Point of Contact

• Title: Clinical Trial Information
• Organization: Millendo Therapeutics

millendo@millendo.com

+1 734-845-9000

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 28, 2018
• First Posted: January 2, 2019
• Study Start: March 25, 2019
• Primary Completion: February 26, 2020
• Study Completion: May 25, 2020
• Last Updated: February 17, 2021
• Results First Posted: February 17, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

ES (4); IT (2); BE (1); US (15); NL (2); GB (3); AU (4); FR (6)