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

NCT ID: NCT03790865

Last Updated: 2021-02-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

158 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-25

Study Completion Date

2020-05-25

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Prader-Willi Syndrome Hyperphagia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Low-Dose Livoletide

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

Group Type EXPERIMENTAL

Livoletide

Intervention Type DRUG

Daily subcutaneous injection

High-Dose Livoletide

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

Group Type EXPERIMENTAL

Livoletide

Intervention Type DRUG

Daily subcutaneous injection

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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Daily subcutaneous injection

Interventions

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Livoletide

Daily subcutaneous injection

Intervention Type DRUG

Placebo

Daily subcutaneous injection

Intervention Type DRUG

Other Intervention Names

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AZP-531

Eligibility Criteria

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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
Minimum Eligible Age

4 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Millendo Therapeutics SAS

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of California - Irvine Medical Center

Orange, California, United States

Site Status

Rady Children's Hospital - San Diego

San Diego, California, United States

Site Status

Children's Hospital Colorado

Denver, Colorado, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Children's Hospitals and Clinics of Minnesota-Minneapolis

Saint Paul, Minnesota, United States

Site Status

Winthrop University Hospital

Mineola, New York, United States

Site Status

New York Presbyterian Morgan Stanley Children's Hospital

New York, New York, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Royal Prince Alfred Hospital

Camperdown, , Australia

Site Status

Austin Health

Melbourne, , Australia

Site Status

Perth Children's Hospital

Nedlands, , Australia

Site Status

The Childrens Hospital at Westmead

Westmead, , Australia

Site Status

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Site Status

Centre Hospitalier Universitaire d'Angers

Angers, , France

Site Status

CHU Lyon - Hopital Femmes Mere Enfant

Bron, , France

Site Status

Hospital Pitie Salpetriere

Paris, , France

Site Status

Hopital Necker-Enfants Malades

Paris, , France

Site Status

CHU de Toulouse - Hospital Rangueil

Toulouse, , France

Site Status

CHU de Toulouse - Hopital des Enfants

Toulouse, , France

Site Status

Azienda Ospedaliera Universitaria Federico II

Napoli, , Italy

Site Status

Ospedale Pediatrico Bambino Gesù

Roma, , Italy

Site Status

Erasmus University Medical Center

Rotterdam, , Netherlands

Site Status

Stichting Kind en Groei

Rotterdam, , Netherlands

Site Status

Hospital General Universitario de Alicante

Alicante, , Spain

Site Status

Hospital de Cruces

Barakaldo, , Spain

Site Status

Hospital Sant Joan de Deu

Barcelona, , Spain

Site Status

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Sabadell, , Spain

Site Status

NHS Tayside

Dundee, , United Kingdom

Site Status

Chelsea and Westminster Hospital

London, , United Kingdom

Site Status

Imperial College London

London, , United Kingdom

Site Status

Countries

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United States Australia Belgium France Italy Netherlands Spain United Kingdom

Provided Documents

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Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

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AZP01-CLI-003

Identifier Type: -

Identifier Source: org_study_id

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