A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome

This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. The study will consist of a Screening Period (up to 28 days), a Treatment Period (28 days), and a Follow-up Period (End-of-Study Visit within 14 days after receiving the last dose of ARD-101). The screening procedures will be initiated upon completion of the informed consent process. Following completion of screening procedures and confirmation of eligibility, subjects will be enrolled to receive ARD-101 in an outpatient setting and will be instructed to visit the clinical center periodically for safety and efficacy assessments.

ARD-101 will be provided as a fixed dose of 200 mg BID for 28 days (Group 1) and then in a dose escalation of 1 week at 400 mg BID, 1 week at 600 mg BID, then 2 weeks at 800 mg BID (Group 2).

Inclusion criteria

• Male and female subjects, 17-65 years of age
• Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
• PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies
• BMI ≥ 18.5 kg/m²
• Qualifying HQ-CT score

Exclusion criteria

• Use of weight loss agents, including herbal medication, within 3 months prior to enrollment
• Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other DSM-III disorders which the investigator believes will interfere significantly with study compliance
• Clinically significant illness in the 8 weeks prior to enrollment
• Current, clinically significant liver, renal, pulmonary, cardiac, oncologic, or gastrointestinal (GI) disease
• Diagnosis of type 1 diabetes mellitus or other active endocrine disorders (e.g., Cushing syndrome, or thyroid dysfunction except if on stable adequate thyroid or glucocorticoid replacement supplement)
• Significant history of abuse of drugs within 1 year prior to enrollment or a positive Drugs of Abuse (DOA) test at screening
• History of alcohol abuse within 1 year prior to enrollment or currently drinks in excess of 21 units per week (3 servings or units/day)