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

Purpose

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

Eligibility

Eligible Ages
Between 4 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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 names:
    • 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 names:
    • 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

More Details

Status
Terminated
Sponsor
Millendo Therapeutics SAS

Study Contact

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.