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

Millendo Therapeutics

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Prader-Willi Syndrome; Hyperphagia

Treatments: Drug: Placebo; Drug: Livoletide

Study type: Interventional

Funder types: Industry

Identifiers

NCT03790865

AZP01-CLI-003

Details and patient eligibility

About

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).

Full 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.

Enrollment: 158 patients

Sex: All

Ages: 4 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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158 participants in 3 patient groups, including a placebo group

Low-Dose Livoletide

Experimental group

Description:

Daily subcutaneous injection of \~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Treatment:

Drug: Livoletide

High-Dose Livoletide

Experimental group

Description:

Daily subcutaneous injection of \~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Treatment:

Drug: Livoletide

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo