A Clinical Study in patients with Prader-Willi-Syndrome (PWS) to test if a study drug named livoletide can reduce food related behaviour and be safe and well tolerated.

A Phase 2b/3 study to evaluate the safety, tolerability, and effects of livoletide (AZP-531), an unacylated ghrelin analog, on food-related behaviors in patients with Prader-Willi syndrome - ZEPHYR

Status

Active, not recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-003062-13-FR

Enrollment

150

Registered

2018-11-21

Start date

2020-02-25

Completion date

Unknown

Last updated

2020-11-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Prader-Willi Syndrome MedDRA version: 20.0 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Livoletide Product Code: AZP-531 Pharmaceutical Form: Solution for injection INN or Proposed INN: Livoletide CAS Number: 1088543-62-7 Concentration unit: mg milligram(s) Concentration ty

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Phase 2b and Phase 3 Core 1. Diagnosis of PWS confirmed by deoxyribonucleic acid (DNA) methylation test. 2. Male and female patients 8 to 65 years of age, inclusive. 3. Have evidence of increased appetite or Hyperphagia Phase 2b and 3 Extension Patients must have participated in the core period. Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 120 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: Core Period Phase 2b 1. History of chronic liver disease, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse. 2. History of significant cardiovascular disease including history of congestive heart failure CHF, New York Heart Association [NYHA] Class 3 or 4), angina pectoris, or myocardial infarction (MI) within 6 months prior to screening. 3. Type 1 diabetes mellitus. 4. Glycated hemoglobin (HbA1c) >10%. 5. History of frequent hypoglycaemia. Phase 3 A patient who enrolled in the Phase 2b part is not eligible for recruitment in the Phase 3 part.

Design outcomes

Primary

Measure	Time frame
Main Objective: Core Period Phase 2b To demonstrate the efficacy of a 3-month treatment with livoletide as compared to placebo for reducing caregiver-observed food-related behavior as assessed by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Phase 3 To demonstrate the efficacy of a 6-month treatment with livoletide as compared to placebo for reducing caregiver-observed food-related behavior as assessed by HQ-CT. ;Secondary Objective: Core Period Phase 2b To demonstrate the efficacy of 3-month treatment with livoletide as compared to placebo for: - Reducing total body fat mass in overweight/obese patients with PWS; - Reducing waist circumference (WC) in overweight/obese patients with PWS; - Reducing body weight (BW) in overweight/obese patients with PWS. Phase 3 To demonstrate the efficacy of 6-month treatment with livoletide as compared to placebo for: - Reducing total body fat mass in overweight/obese patients with PWS; - Reducing WC in overweight/obese patients with PWS; - Reducing BW in overweight/obese patients with PWS. Extension period: - Maintenance of effects - Pharmacokinetics;Primary end point(s): Core Period Phase 2b • Change from baseline to the end of the 3-month Core Period in HQ-CT total score. Phase 3 • Change from baseline to the end of the 6-month Core Period in HQ-CT total score.;Timepoint(s) of evaluation of this end point: Phase 2b - 3 months Phase 3 - 6 months	—

Measure

Time frame

Main Objective: Core Period Phase 2b To demonstrate the efficacy of a 3-month treatment with livoletide as compared to placebo for reducing caregiver-observed food-related behavior as assessed by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Phase 3 To demonstrate the efficacy of a 6-month treatment with livoletide as compared to placebo for reducing caregiver-observed food-related behavior as assessed by HQ-CT. ;Secondary Objective: Core Period Phase 2b To demonstrate the efficacy of 3-month treatment with livoletide as compared to placebo for: - Reducing total body fat mass in overweight/obese patients with PWS; - Reducing waist circumference (WC) in overweight/obese patients with PWS; - Reducing body weight (BW) in overweight/obese patients with PWS. Phase 3 To demonstrate the efficacy of 6-month treatment with livoletide as compared to placebo for: - Reducing total body fat mass in overweight/obese patients with PWS; - Reducing WC in overweight/obese patients with PWS; - Reducing BW in overweight/obese patients with PWS. Extension period: - Maintenance of effects - Pharmacokinetics;Primary end point(s): Core Period Phase 2b • Change from baseline to the end of the 3-month Core Period in HQ-CT total score. Phase 3 • Change from baseline to the end of the 6-month Core Period in HQ-CT total score.;Timepoint(s) of evaluation of this end point: Phase 2b - 3 months Phase 3 - 6 months

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Secondary

Measure	Time frame
Secondary end point(s): Core Period Phase 2b • Percentage change from baseline to the end of the 3-month Core Period in total body fat mass in overweight/obese patients with PWS; • Change from baseline to the end of the 3-month Core Period in waist circumference in overweight/obese patients with PWS; • Percentage change from baseline to the end of the 3-month Core Period in body weight in overweight/obese patients with PWS Phase 3 • Percentage change from baseline to the end of the 6-month Core Period in total body fat mass in overweight/obese patients with PWS; • Change from baseline to the end of the 6-month Core Period in waist circumference in overweight/obese patients with PWS; • Percentage change from baseline to the end of the 6-month Core Period in body weight in overweight/obese patients with PWS.;Timepoint(s) of evaluation of this end point: Phase 2b - 3 months Phase 3 - 6 months	—

Measure

Time frame

Secondary end point(s): Core Period Phase 2b • Percentage change from baseline to the end of the 3-month Core Period in total body fat mass in overweight/obese patients with PWS; • Change from baseline to the end of the 3-month Core Period in waist circumference in overweight/obese patients with PWS; • Percentage change from baseline to the end of the 3-month Core Period in body weight in overweight/obese patients with PWS Phase 3 • Percentage change from baseline to the end of the 6-month Core Period in total body fat mass in overweight/obese patients with PWS; • Change from baseline to the end of the 6-month Core Period in waist circumference in overweight/obese patients with PWS; • Percentage change from baseline to the end of the 6-month Core Period in body weight in overweight/obese patients with PWS.;Timepoint(s) of evaluation of this end point: Phase 2b - 3 months Phase 3 - 6 months

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Countries

Belgium, Canada, France, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

Public ContactClinical Trial Information

Millendo Therapeutics SAS

zephyr@millendo.com+33 472 18 09 30

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 21, 2026