Prader-Willi Syndrome MedDRA version: 17.1 Level: HLGT Classification code 10003018 Term: Appetite and general nutritional disorders System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 17.1 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1 Level: HLT Classification code 10003022 Term: Appetite disorders System Organ Class: 10027433 - Metabolism and nutr
Conditions
Interventions
Product Name: AZP-531
Product Code: AZP-531
Pharmaceutical Form: Lyophilisate for solution for injection
INN or Proposed INN: INN Not yet proposed
Current Sponsor code: AZP-531
Concentration unit: mg
Sponsors
Alize Pharma
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Male and female patients with genetically confirmed diagnosis of Prader-Willi Syndrome using standard DNA methylation test or fluorescent in situ hybridization -Patients aged 18 years to 40 years old inclusive initially o Patients aged 12 years old to less than 18 years old after formal decision of the Data Monitoring Committee -Have evidence of increased appetite or hyperphagia, as judged by the investigator -Patients on well-balanced controlled diet and on a regular exercise program as recommended in common clinical practice for this patient population -Women of Child Bearing Potential (WCBP) must have a negative pregnancy test on admission to the study center -All WCBP, sexually active male subjects, and all partners of subjects should agree to use adequate methods of birth control (diaphragm, condoms with spermicide?) throughout the study and for 30 days after the last dose of study drug -Adequate renal function, defined as serum creatinine inferior or equal to 1.5 x Upper Limit of Normal (ULN) and urine protein/creatinine ratio =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -History of chronic liver disease, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse - History of acute or chronic pancreatitis -Type 1 diabetes -Insulin treatment -Use of weight loss agents or drugs known to affect appetite (including GLP-1 analogs) within 2 months prior to screening -Co-morbid condition or disease (such as respiratory disease or psychiatric disorder) diagnosed less than 1 month prior to screening -Co-morbid condition or disease or abnormal laboratory finding that would in the investigator judgment increase the subject risk to participating in this study and that will not allow the patient to complete the study -History or presence of gastrointestinal, hepatic or renal disease or any other condition known to interfere with the absorption, distribution, metabolism or excretion of drugs -Participation in a clinical trial with an investigational agent within 2 months prior to screening -Clinically significant abnormalities on ECG at screening -Pregnant or lactating woman -History of hypersensitivity to drugs with a similar chemical structure or class as AZP-531 (Acylated Ghrelin and Unacylated Ghrelin) -Unwillingness or inability to follow the procedures outlined in the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of one dose of AZP-531 adjusted for bodyweight (2, 3 or 4 mg) administered over 2 weeks as daily subcutaneous injection.;Secondary Objective: To evaluate effects of AZP-531 after 2 weeks of administration on fullness/satiety and appetite/prospective food consumption as assessed by Numerical Rating Scales (NRS).;Primary end point(s): Evaluation of safety will be based on patients who have received at least one dose of study medication. Individual and summary statistics including mean, median, standard-deviation (SD) and range will be presented in tabular form by treatment group for vital signs and clinical laboratory data. Adverse events will be tabulated and summarised according to the current version of the Medical Dictionary for Regulatory Activities (MedDRA) and presented using the total number of AEs and the total number and percent of patients who experienced an AE per treatment group.;Timepoint(s) of evaluation of this end point: AEs will be collected from the signature of informed consent. Safety laboratory evaluation will be performed at screening (D-2) and at the end of treatment period (D14) Vital signs will be obtained at screening (D-2), Baseline (D-1) and at the end of treatment period (D14) Waist Circumference, Weight, Height and BMI will be measured (derived from weight and height for BMI) at Baseline (D-1) and at the end of treatment period (D14) except for Height (only D-1) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The Numeric Rating Scales: fullness/satiety and appetite/prospective food consumption will be presented descriptively by time (before meal, just after meal, 120 minutes after meal), meal (breakfast, lunch), visit (D-1, D1, D14) and treatment. Analysis of covariance (ANCOVA) or analysis of variance (ANOVA) will be performed at Day 14 for the afforded mentioned parameters. The model will include the parameter at baseline (when appropriate), the stratification variables and the treatment group. Key interactions will also be included to explore subgroup effect (e.g. Age Group-by-Treatment, Genetic Subtype-by-Treatment).;Timepoint(s) of evaluation of this end point: Numerical Rating Scales will be administered to patients at baseline (D-1), first day of treatment (D1) and at the end of treatment period (D14) | — |
Countries
France, Italy, Spain
Contacts
Public ContactMedical Director
Alizé Pharma
Outcome results
None listed