A Phase IIa study to evaluate the safety, tolerability, and effects of AZP-531 on food-related behavior in patients with Prader-Willi Syndrome

Phase 1

• Conditions: Prader-Willi Syndrome; MedDRA version: 17.1Level: HLGTClassification code 10003018Term: Appetite and general nutritional disordersSystem Organ Class: 10027433 - Metabolism and nutrition disorders; MedDRA version: 17.1Level: PTClassification code 10036476Term: Prader-Willi syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 17.1Level: HLTClassification code 10003022Term: Appetite disordersSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2014-001670-34-ES
• Lead Sponsor: Alize Pharma

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10-24
• Last Update Posted: 9 May 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

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 <0.2
-Adequate hepatic function, defined as total bilirubin inferior or equal to 1.5 x ULN and Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) levels inferior or equal to 3 x ULN
-Growth hormone treatment will be permitted if doses have been stable for at least 1 month prior to screening
-Psychotropic treatment will be permitted and should be stable at least 1 month prior to screening
-Any other treatment including thyroid hormones should be stable for at least 1 month prior to screening
-Informed consent signed by the patient, parent or legal guardian, as appropriate
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified