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

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

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233
Contact:
Hussein Abdul-Latif, MD

More Details

NCT ID
NCT03790865
Status
Recruiting
Sponsor
Millendo Therapeutics SAS

Study Contact

Nadège Tremel
+33 472 18 09 30
tremel@millendo.com

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.