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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

Phase 2
Completed
Conditions
Prader-Willi syndrome
PWS
10027424
10003018
10014067
Registration Number
NL-OMON48225
Lead Sponsor
Millendo Therapeutics SAS
Brief Summary

Trial ended prematurely

Detailed Description

Not available

Recruitment & Eligibility

Status
Completed
Sex
Not specified
Target Recruitment
23
Inclusion Criteria

- A confirmed gentic diganosis of PWS. Documentation of PWS subtype. If PWS
subtype is not known, a sample for testing may be obtained
and the patient may continue on to be enrolled into the study if he/she meets
all the other inclusion criteria and non of the
exclusion criteria.

- Male and female patients 4 to 65 years of age.

- Evidence of increased appetite or hyperphagia, as jugded by the investigator
and HQ-CT score.

- Patient must have a single primary caregiver who should be available for the
duration of the study. On average,
approximately 4 (or more) waking hours per day with the patient.

- BMI * 65 kg/m2

- Growth hormone treatment permitted if doses are stable

Exclusion Criteria

- History of chronic liver disease
- Type 1 diabetes mellitus
- HbA1c > 10%

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>Primary Outcome Measures :<br /><br>Change in hyperphagia and food-related behaviors (Hyperphagia Questionnaire for<br /><br>Clinical Trials; HQ-CT) [ Time Frame: Baseline to month 3 ]<br /><br><br /><br>Change from baseline to the end of the 3-month Core Period for HQ-CT total<br /><br>score. The HQ-CT score range is 0 to 36 where the higher score represents more<br /><br>severe abnormal food related behaviors.</p><br>
Secondary Outcome Measures
NameTimeMethod
<p>Secondary Outcome Measures :<br /><br>Change in fat mass [ Time Frame: Baseline to month 3 ]<br /><br>Percentage change from baseline to the end of the 3-month Core Period in total<br /><br>body fat mass in overweight/obese patients with PWS<br /><br><br /><br>Change in waist circumference [ Time Frame: Baseline to month 3 ]<br /><br>Change from baseline to the end of the 3-month Core Period in waist<br /><br>circumference in overweight/obese patients with PWS<br /><br><br /><br>Change in body weight [ Time Frame: Baseline to month 3 ]<br /><br>Percentage change from baseline to the end of the 3-month Core Period in body<br /><br>weight in overweight/obese patients with PWS</p><br>
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