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).
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
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
158
Daily subcutaneous injection
Daily subcutaneous injection
Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT)
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.
Time frame: Baseline to month 3
Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese
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
Time frame: Baseline to month 3
Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese
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
Time frame: Baseline to month 3
Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese
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
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University of Alabama at Birmingham
Birmingham, Alabama, United States
University of California - Irvine Medical Center
Orange, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Children's Hospitals and Clinics of Minnesota-Minneapolis
Saint Paul, Minnesota, United States
Winthrop University Hospital
Mineola, New York, United States
...and 27 more locations
Time frame: Baseline to month 3