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

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

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

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: ARD-101

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.

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).

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 17 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ARD-101 (Fixed Dose); ARD-101: 4 weeks dosing at 200 mg BID (twice daily)	Drug: ARD-101; Oral administration of ARD-101 taken BID (twice daily) for 28 days.
Experimental: ARD-101 (Dose Escalation); ARD-101: 1 week at 400 mg BID (twice daily), second week at 600 mg BID, third and fourth weeks at 800 mg BID. Oral administration.	Drug: ARD-101; Oral administration of ARD-101 taken BID (twice daily) for 28 days.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment Emergent Adverse Events (TEAE)	The incidence of treatment emergent adverse events (TEAE) reported through the 28 days of treatment and the 14 days post treatment phase. TEAEs were those events occurring after treatment began.	Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up).

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome	Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). The HQCT is a 9-item hyperphasia questionnaire which is validated for use in PWS clinical trials. Score will range from 0 (no hyperphagia behaviors) to 36 (most severe hyperphagia behaviors)	Baseline, Day 15, Day 28
Change in Body Weight	Total weight change at the end of treatment (day 28) from baseline	Baseline to day 28

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect on Anxiousness	Evaluation of patient's anxiousness via the PWS anxiousness and distress questionnaire (PADQ) obtained at study visits.	Baseline to Day 28
The Change in Body Composition	The change in body composition based on evaluation of dual-energy X-ray absorptiometry (DEXA) scans at the end of treatment compared to baseline	Baseline to Day 28
Effect on Psychiatric Status	Effect on psychiatric status through screening presence of suicidal ideation and behavior in addition to screening for degree of depression via the Columbia-Suicide Severity Rating Scale (C-SSRS), as assessed by caregiver.	Baseline to Day 28
The Change in Body-Mass Index (BMI)	The change in body-mass index (BMI) at the end of treatment from the baseline as well as 28 days after end of treatment	Baseline to Day 28
The Change in Waist Circumference	The change in waist circumference at the end of treatment from the baseline as well as 14 days after end of treatment	Baseline to Day 28
Change in Patient Health	Difference from Day 28 to baseline in patient health as assessed by Patient Health Questionnaire ((PHQ)-9 Questionnaires)	Baseline to Day 28
Change in Body Fat	Estimation of body fat by bioelectric impedance analysis at the end of treatment compared to baseline	Baseline to Day 28
Change in GI Passage	Change in GI passage time to explore potentially reduced constipation	Baseline to Day 28
Effect on Psychiatric Status	Effect on psychiatric status as assessed by Patient Health Questionnaire ((PHQ)-9 Questionnaires)	Baseline to Day 28

Collaborators and Investigators

• Sponsor: Aardvark Therapeutics, Inc.
• Collaborators: Children's Hospital Colorado; Stanford University
• Investigators: Principal Investigator: Shawn McCandless, MD, Children's Hospital Colorado

Study record dates

Study Major Dates

• Study Start (Actual): May 27, 2022
• Primary Completion (Actual): September 24, 2024
• Study Completion (Actual): September 24, 2024

Study Registration Dates

• First Submitted: November 30, 2021
• First Submitted That Met QC Criteria: November 30, 2021
• First Posted (Actual): December 10, 2021

Study Record Updates

• Last Update Posted (Actual): September 8, 2026
• Last Update Submitted That Met QC Criteria: September 3, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Imprinting Disorders; Neurologic Manifestations; Nervous System Diseases; Nutrition Disorders; Genetic Diseases, Inborn; Overnutrition; Neurobehavioral Manifestations; Congenital Abnormalities; Abnormalities, Multiple; Overweight; Intellectual Disability; Obesity; Chromosome Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Prader-Willi Syndrome
• Other Study ID Numbers: AARD-203

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No