Summary

Eligibility
for people ages 4-65 (full criteria)
Location
at Orange, California and other locations
Dates
study started
estimated completion

Description

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).

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

Details

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.

Keywords

Prader-Willi Syndrome Hyperphagia PWS Syndrome Livoletide

Eligibility

You can join if…

Open to people ages 4-65

  • 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 CAN'T join if...

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

Locations

  • University of California - Irvine Medical Center
    Orange California 92868 United States
  • Rady Children's Hospital - San Diego
    San Diego California 92123 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Millendo Therapeutics SAS
ID
NCT03790865
Phase
Phase 2/3
Study Type
Interventional
Last Updated