Clinical trial NCT03790865: Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Prader-Willi Syndrome
Hyperphagia
Keywords: Prader-Willi Syndrome
PWS
Lead sponsor: Millendo Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-01-02
Enrollment: 158
Sex: ALL
Minimum age (years): 4
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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.

Eligibility 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

Primary outcomes:
measure: Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT)
timeFrame: Baseline to month 3
Secondary outcomes:
measure: Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese
timeFrame: Baseline to month 3
measure: Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese
timeFrame: Baseline to month 3
measure: Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese
timeFrame: Baseline to month 3
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Orange
country: United States
facility: University of California - Irvine Medical Center
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital - San Diego
state: California
city: Denver
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Saint Paul
country: United States
facility: Children's Hospitals and Clinics of Minnesota-Minneapolis
state: Minnesota
city: Mineola
country: United States
facility: Winthrop University Hospital
state: New York
city: New York
country: United States
facility: New York Presbyterian Morgan Stanley Children's Hospital
state: New York
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
city: Melbourne
country: Australia
facility: Austin Health
city: Nedlands
country: Australia
facility: Perth Children's Hospital
city: Westmead
country: Australia
facility: The Childrens Hospital at Westmead
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Angers
country: France
facility: Centre Hospitalier Universitaire d'Angers
city: Bron
country: France
facility: CHU Lyon - Hopital Femmes Mere Enfant
city: Paris
country: France
facility: Hospital Pitie Salpetriere
city: Paris
country: France
facility: Hopital Necker-Enfants Malades
city: Toulouse
country: France
facility: CHU de Toulouse - Hospital Rangueil
city: Toulouse
country: France
facility: CHU de Toulouse - Hopital des Enfants
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Rotterdam
country: Netherlands
facility: Erasmus University Medical Center
city: Rotterdam
country: Netherlands
facility: Stichting Kind en Groei
city: Alicante
country: Spain
facility: Hospital General Universitario de Alicante
city: Barakaldo
country: Spain
facility: Hospital de Cruces
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Sabadell
country: Spain
facility: Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
city: Dundee
country: United Kingdom
facility: NHS Tayside
city: London
country: United Kingdom
facility: Chelsea and Westminster Hospital
city: London
country: United Kingdom
facility: Imperial College London
Study officials:
References: