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

NCT05153434 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: AARD-203
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 2

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

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events (TEAE)

  The incidence of treatment-emergent adverse events (TEAE) during the treatment period

  Baseline to Day 28

Secondary Outcomes (2)

• Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome

  Baseline, Day 15, Day 28

• Effect on Weight

  Baseline to Day 28

Other Outcomes (9)

• Effect on Anxiousness

  Baseline to Day 28

• The Change in Body Composition

  Baseline to Day 28

• Effect on Psychiatric Status

  Baseline to Day 28

Study Arms (1)

ARD-101

EXPERIMENTAL

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.

Drug: ARD-101

Interventions

ARD-101 DRUG

Twice Daily, Oral Administration

ARD-101

Eligibility Criteria

• Age: 17 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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):
• mmHg \<systolic blood pressure (SBP) \<160 mmHg
• mmHg \<diastolic blood pressure (DBP) \<100 mmHg
• bpm \<heart rate (HR) \<100 bpm
• Stable body weight for \~2 months (self or guardian-reported loss/gain within ± 10%) prior to enrollment
• Standard 12-lead ECG parameters after 10 minutes resting in supine position in the following ranges; 120 ms \<PR \<220 ms, QRS \<120 ms, QTc ≤430 ms if male, ≤450 ms if female and normal ECG tracing unless the Investigator considers an ECG abnormality to be not clinically relevant.

You may not qualify if:

• 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

Sponsors & Collaborators

• Aardvark Therapeutics, Inc.: lead
• Children's Hospital Colorado: collaborator
• Stanford University: collaborator

Study Sites (2)

Stanford University

Palo Alto, California, 94304, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2021
• First Posted: December 10, 2021
• Study Start: May 27, 2022
• Primary Completion: September 24, 2024
• Study Completion: September 24, 2024
• Last Updated: May 9, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)