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

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ClinicalTrials.gov Identifier: NCT03790865
Recruitment Status : Terminated (Livoletide did not result in a statistically significant improvement in hyperphagia and food-related behaviors)
First Posted : January 2, 2019
Results First Posted : February 17, 2021
Last Update Posted : February 17, 2021
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc. ( Millendo Therapeutics SAS )

Tracking Information
First Submitted Date  ICMJE December 28, 2018
First Posted Date  ICMJE January 2, 2019
Results First Submitted Date  ICMJE January 21, 2021
Results First Posted Date  ICMJE February 17, 2021
Last Update Posted Date February 17, 2021
Actual Study Start Date  ICMJE March 25, 2019
Actual Primary Completion Date February 26, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 11, 2019)
Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT) [ Time Frame: Baseline to month 3 ]
Change from baseline to the end of the 3-month Core Period for HQ-CT total score. The HQ-CT score range is 0 to 36 where the higher score represents more severe abnormal food related behaviors.
Original Primary Outcome Measures  ICMJE
 (submitted: December 28, 2018)
Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT) [ Time Frame: Baseline to month 3 ]
Change from baseline to the end of the 3-month Core Period for HQ-CT total score
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 8, 2021)
  • Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]
    Total body fat mass was assessed using dual energy X-ray absorptiometry (DXA) scan. DXAs were conducted at each local facility using standardized procedures and settings. Overweight/obese patients were defined as follows:
    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
  • Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]
    The waist circumference was measured in fasting condition at the superior border of iliac crest, according to recommendations from the Anthropometry Procedures Manual of the National Health and Nutrition Examination Survey, Revised 2007. Overweight/obese patients were defined as follows:
    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
  • Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese [ Time Frame: Baseline to month 3 ]
    Patients were weighed in fasting condition, clothed (underwear, light gown or light clothing), without footwear or heavy jewelry, using a calibrated scale. The same scale should be used throughout the study if possible. The conditions under which patients are weighed should be kept consistent if possible. Overweight/obese patients were defined as follows:
    • patients ≥18 years of age: BMI ≥27 kg/m2
    • patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
Original Secondary Outcome Measures  ICMJE
 (submitted: December 28, 2018)
  • Change in fat mass [ Time Frame: Baseline to month 3 ]
    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 in waist circumference [ Time Frame: Baseline to month 3 ]
    Change from baseline to the end of the 3-month Core Period in waist circumference in overweight/obese patients with PWS
  • Change in body weight [ Time Frame: Baseline to month 3 ]
    Percentage change from baseline to the end of the 3-month Core Period in body weight in overweight/obese patients with PWS
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
Official Title  ICMJE 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
Brief 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).
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Prader-Willi Syndrome
  • Hyperphagia
Intervention  ICMJE
  • Drug: Livoletide
    Daily subcutaneous injection
    Other Name: AZP-531
  • Drug: Placebo
    Daily subcutaneous injection
Study Arms  ICMJE
  • 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.
    Intervention: Drug: Livoletide
  • 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.
    Intervention: Drug: Livoletide
  • 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.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 6, 2020)
158
Original Estimated Enrollment  ICMJE
 (submitted: December 28, 2018)
150
Actual Study Completion Date  ICMJE May 25, 2020
Actual Primary Completion Date February 26, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   France,   Italy,   Netherlands,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03790865
Other Study ID Numbers  ICMJE AZP01-CLI-003
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Millendo Therapeutics, Inc. ( Millendo Therapeutics SAS )
Study Sponsor  ICMJE Millendo Therapeutics SAS
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Millendo Therapeutics, Inc.
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP