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

Trial ID or NCT#

NCT05153434

Status

recruiting iconRECRUITING

Purpose

A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome

Official Title

A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome

Eligibility Criteria

Ages Eligible for Study: 17 Years to 65 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * 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:
    1. 1. 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. 2. Fasting plasma glucose \<140 mg/dL during the Screening Period 3. 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):
    2. 1. 95 mmHg \3 months) of medications commonly used in PWS patients are allowed
Exclusion Criteria:
  1. * 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)* 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)* Caffeine consumption exceeding 6 cups of caffeinated tea/coffee (or equivalent) per day* Participation in any clinical study with an investigational drug/device within 1 month prior to enrollment* Serious adverse reaction or significant hypersensitivity to any drug* Clinically significant blood loss or blood donation \> 500 mL within 3 months prior to enrollment* Inadequate venous access* History of significant drug hypersensitivity or anaphylaxis* Any condition that the investigator or primary physician believes may not be appropriate for participating the study
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Investigator(s)

Diane Stafford

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Contact

Diane Stafford, MD
650-721-1811

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