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
| Arm | Description | Assigned 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. |
|
|
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. |
|
|
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. |
|
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.