A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome
Overview
- Phase
- Phase 2
- Intervention
- ARD-101
- Conditions
- Prader-Willi Syndrome
- Sponsor
- Aardvark Therapeutics, Inc.
- Enrollment
- 19
- Locations
- 2
- Primary Endpoint
- Incidence of Treatment-Emergent Adverse Events (TEAE)
- Status
- Completed
- Last Updated
- 11 months ago
Overview
Brief Summary
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
Detailed Description
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.
Investigators
Eligibility Criteria
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
- •A HQ-CT score ≥ 10
- •If a subject has a diagnosis of type 2 diabetes, the following criteria must be met:
- •Hemoglobin A1c (HbA1c) \<7.5% not being managed with insulin. Patients taking glucagon-like peptide (GLP)-1 analogues (exenatide or liraglutide) must have been on stable dose for greater than 3 months.
- •Fasting plasma glucose \<140 mg/dL during the Screening Period
- •No history of ketoacidosis or hyperosmolar coma
- •Stable or well-controlled blood pressure (BP) and vital signs. Specifically: Vital signs after 5 minutes resting in seated position (feet flat on floor, back supported):
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
- •A PHQ-9 score of ≥10
- •Any suicidal ideation of type 4 or 5 on the C-SSRS
- •Clinically significant illness in the 8 weeks prior to enrollment
- •History of clinically significant bleeding disorders
- •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)
- •Liver disease or liver injury as indicated by abnormal liver function tests, SGOT (aspartate aminotransferase (AST)), alkaline phosphatase, or serum bilirubin (\> 1.5 x upper limit of normal (ULN) for any of these tests) or history of hepatic cirrhosis
- •History or presence of impaired renal function as indicated by clinically significantly abnormal creatinine, blood urea nitrogen (BUN), or urinary constituents (e.g., albuminuria) or moderate to severe renal dysfunction as defined by the Cockroft Gault equation (\< 50 mL/min)
Arms & Interventions
ARD-101
First week 400 mg of ARD-101 twice daily, second week 600 mg of ARD-101 twice daily, third week 800 mg of ARD-101 twice daily, fourth week 800 mg of ARD-101 twice daily.
Intervention: ARD-101
Outcomes
Primary Outcomes
Incidence of Treatment-Emergent Adverse Events (TEAE)
Time Frame: Baseline to Day 28
The incidence of treatment-emergent adverse events (TEAE) during the treatment period
Secondary Outcomes
- Effect on Weight(Baseline to Day 28)
- Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome(Baseline, Day 15, Day 28)