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

Phase 3

Recruiting

• Conditions: Hyperphagia; Prader-Willi Syndrome
• Interventions: Drug: Placebo; Drug: ARD-101

• Registration Number: NCT06828861
• Lead Sponsor: Aardvark Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-12-20
• Study First Submitted: 2025-02-13
• Study First Submitted QC: 2025-02-13
• Study First Posted: 2025-02-17
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-21
• Last Update Posted: 2025-05-23
• Primary Completion: 2026-03
• Study Completion: 2026-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

• Documented confirmation of Prader-Willi Syndrome (PWS)
• Stable care setting with same, single designated caregiver for at least 6 months prior to Visit 1

Exclusion Criteria

• 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 glucocorticoids: oral, intra-articular, or intravenous

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment Arm B	Placebo	Placebo for ARD-101
Treatment Arm A	ARD-101	ARD-101

Primary Outcome Measures

Name	Time	Method
Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score	Baseline to Week 12	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).

Secondary Outcome Measures

Name	Time	Method
Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients	Baseline to Week 12	The CaGI-S is a single-item, 7-point scale to describe the severity of the PWS patient's hyperphagia (excessive hunger). It is completed by the patient's caregiver. The minimum score is 1 (not present) and the maximum score is 7 (extremely severe). A higher score indicates a worse severity of hyperphagia.
Change in Clinical Global Impression of Severity (CGI-S) Score for Hyperphagia in Prader-Willi patients	Baseline to Week 12	The CGI-S is a single-item, 7-point scale designed to assess the severity of the PWS patient's hyperphagia (excessive hunger). It is assessed by the clinician and considers the clinician's experience with the PWS population. The minimum score is 1 (normal, not at all ill) and the maximum score is 7 (among the most extremely ill patients). A higher score indicates a worse severity of hyperphagia.

Trial Locations

Locations (42)

Children's of Alabama; 🇺🇸 Birmingham, Alabama, United States

Rady Children's Hospital; 🇺🇸 Encinitas, California, United States

Stanford Childrens Health Specialty Services; 🇺🇸 Palo Alto, California, United States

Childrens Hospital Colorado; 🇺🇸 Denver, Colorado, United States

Nemours Children Clinic Wilmington; 🇺🇸 Wilmington, Delaware, United States

UF Shands Childrens Hospital; 🇺🇸 Gainesville, Florida, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

University of Minnesota Masonic Children's Hospital; 🇺🇸 Minneapolis, Minnesota, United States

Maimonides Medical Center; 🇺🇸 Brooklyn, New York, United States

NYU Langone Children's Ambulatory Care Center; 🇺🇸 Mineola, New York, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Cook Children's Medical Center; 🇺🇸 Fort Worth, Texas, United States

Texas Valley Clinical Research, LLC; 🇺🇸 Weslaco, Texas, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

Royal Prince Alfred Hospital; 🇦🇺 Camperdown, New South Wales, Australia

John Hunter Children's Hospital; 🇦🇺 New Lambton, New South Wales, Australia

The Children's Hospital at Westmead; 🇦🇺 Westmead, New South Wales, Australia

Queensland Children's Hospital; 🇦🇺 South Brisbane, Queensland, Australia

Alberta Children's Hospital Research Institute; 🇨🇦 Calgary, Alberta, Canada

Li Ka Shing Centre for Health Research Innovation; 🇨🇦 Edmonton, Alberta, Canada

Centre Hospitalier Universitaire Sainte-Justine; 🇨🇦 Montreal, Quebec, Canada

CHU de Toulouse-Hôpital Des Enfants; 🇫🇷 Toulouse, Haute-Garonne, France

CHU d'Angers; 🇫🇷 Angers, Maine-et-Loire, France

AP-HP - Hôpital de la Pitié Salpétrière; 🇫🇷 Paris, France

AP-HP - Hôpital universitaire Necker-Enfants malades; 🇫🇷 Paris, France

CHU de Toulouse- Hôpital de Rangueil; 🇫🇷 Toulouse, France

Ospedale Pediatrico Bambino Gesù IRCCS; 🇮🇹 Roma, Lazio, Italy

Ospedale San Raffaele S.r.l. - PPDS; 🇮🇹 Milano, Lombardia, Italy

Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN; 🇮🇹 Firenze, Toscana, Italy

Inha University Hospital; 🇰🇷 Incheon, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of

Ajou University Hospital; 🇰🇷 Suwon-si, Korea, Republic of

National University Hospital for Children's Neurorehabilitation "Dr. Nicolae Robanescu"; 🇷🇴 Bucharest, Bucuresti, Romania

Institutul National de Endocrinologie C. I. Parhon; 🇷🇴 Bucuresti, Romania

Spitalul Clinic de Urgenta Sfantul Spiridon; 🇷🇴 Iaşi, Romania

Corporacio Sanitaria Parc Tauli; 🇪🇸 Sabadell, Barcelona, Spain

Hospital Universitario Vall d'Hebron - PPDS; 🇪🇸 Barcelona, Spain

Hospital Universitario 12 de Octubre; 🇪🇸 Madrid, Spain

Leicester Royal Infirmary; 🇬🇧 Leicester, Leicestershire, United Kingdom

Fulborn Hospital; 🇬🇧 Cambridge, United Kingdom

Royal Hospital for Children (Glasgow) - PPDS - PIN; 🇬🇧 Glasgow, United Kingdom

The Royal London Hospital; 🇬🇧 London, United Kingdom