ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial

NCT06828861 | Suspended Phase 3 DMC FDA Drug

• 1 other identifier: AVK-101-301
• Study Type: interventional
• Target: 90
• Locations: 5 countries
• Sites: 30

Brief Summary

The goal of this clinical trial is to learn if ARD-101 works to treat hyperphagia-related behavior in patients with Prader-Willi syndrome (PWS). It will also teach us about the safety of ARD-101. The main questions it aims to answer are:

• Does ARD-101 improve the total score of the HQCT-9 (hyperphagia questionnaire for clinical trials, 9 questions)?
• What medical problems do participants have when taking ARD-101? Researchers will compare ARD-101 to a placebo (a look-alike substance that contains no drug) to see if ARD-101 works to treat hyperphagia in PWS subjects. Eligible participants will:
• Take ARD-101 or a placebo every day for 12 weeks.
• Visit the clinic or have a tele-visit once every 2 to 4 weeks during dosing and then have a tele-visit 4 weeks after stopping the ARD-101 or placebo.
• Patients/Caregivers will keep a daily diary. Participants who complete the study may be eligible to enter an open-label extension study where everyone will receive ARD-101.

Conditions

Hyperphagia; Prader-Willi Syndrome

Keywords

Prader-Willi Syndrome; ARD-101; PWS; Prader-Willi; Prader Willi; Prader Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score

  The HQ-CT score is a 9 question, 5-point scale to describe the PWS patient's hyperphagia food-related problem behaviors. It is completed by the patient's caregiver. Each question is scored from 0 to 4. The minimum total score is 0 (hyperphagia related behavior symptoms not exhibited) and the maximum total score is 36 (hyperphagia related behavior symptoms are observed).

  Baseline to Week 12

Secondary Outcomes (2)

• Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients

  Baseline to Week 12

• Change in Clinical Global Impression of Severity (CGI-S) Score for Hyperphagia in Prader-Willi patients

  Baseline to Week 12

Study Arms (2)

Treatment Arm A

EXPERIMENTAL

ARD-101

Drug: ARD-101

Treatment Arm B

PLACEBO COMPARATOR

Placebo for ARD-101

Drug: Placebo

Interventions

Placebo DRUG

200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 10 weeks

Treatment Arm B

ARD-101 DRUG

200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 10 weeks

Treatment Arm A

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented confirmation of Prader-Willi Syndrome (PWS)
• Stable care setting with same, single designated caregiver for at least 6 months prior to Visit 1
• At least 7 years of age or older in the US at the time of consent
• At least 10 years of age or older in Australia
• At least 13 years of age or older in countries outside of the US and Australia

You may not qualify if:

• Diagnosis of schizophrenia, bipolar disorder, personality disorder or other severe mood, anxiety or eating disorder (other than hyperphagia).
• Presence of any malignancy within 5 years with the exception of basal or squamous cell carcinoma of the skin, in situ carcinoma of the service, or in situations prostate cancer.
• Presence of clinically relevant renal, hepatic, pancreatic, cardiovascular, neurological, psychiatric, hematological, pulmonary, or GI abnormality that, in the opinion of the investigator, may preclude the patient from safe completion of the study
• Adults: systolic blood pressure \>=160 mmHg and/or diastolic blood pressure \>=100 mmHg
• Children and Adolescents: systolic blood pressure \>=140 mmHg and/or diastolic blood pressure \>=90 mmHg.
• Type 1 diabetes mellitus; HbA1c \>8.5%
• Use of agents to promote weight gain or loss, alter hunger or appetite within 30 days of Visit 1 and throughout the study.
• Use of any commercially available medication for the treatment of hyperphagia (i.e., Vykat) within 60 days of randomization and throughout the study.
• Very high doses of glucocorticoids in the previous 3 months of Visit 1 and throughout the study.

Sponsors & Collaborators

• Aardvark Therapeutics, Inc.: lead

Study Sites (30)

Children's of Alabama

Birmingham, Alabama, 35233-1711, United States

Location

Rady Children's Hospital

Encinitas, California, 92123, United States

Location

Children's Hospital of Orange Country

Orange, California, 92868, United States

Location

Stanford Children's Health Specialty Services

Palo Alto, California, 94304, United States

Location

Children's Hospital Colorado

Denver, Colorado, 80045-7106, United States

Location

Nemours Children Clinic Wilmington

Wilmington, Delaware, 19803-3607, United States

Location

UF Shands Children's Hospital

Gainesville, Florida, 32610-3008, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322-1047, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611-2991, United States

Location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287-0005, United States

Location

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, 55102, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11219-2918, United States

Location

NYU Langone Children's Ambulatory Care Center

Mineola, New York, 11501-4077, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-0005, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia

Location

Alberta Children's Hospital Research Institute

Calgary, Alberta, T3B 6A8, Canada

Location

Stollery Children's Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

Children's Hospital at London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Inha University Hospital

Incheon, 22332, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Ajou University Hospital

Suwon, 16499, South Korea

Location

Leicester Royal Infirmary

Leicester, Leicestershire, LE1 5WW, United Kingdom

Location

Fulborn Hospital

Cambridge, CB21 5EF, United Kingdom

Location

Royal Hospital for Children (Glasgow) - PPDS - PIN

Glasgow, G51 4TF, United Kingdom

Location

The Royal London Hospital

London, E1 4NS, United Kingdom

Location

MeSH Terms

Conditions

Hyperphagia; Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; 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 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 13, 2025
• First Posted: February 17, 2025
• Study Start: December 20, 2024
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): April 1, 2027
• Last Updated: April 9, 2026

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

KR (3); GB (4); AU (3); US (16); CA (4)