Clinical trial NCT06828861: ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of ARD-101 for the Treatment of Hyperphagia in Patients With Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hyperphagia
Prader-Willi Syndrome
Keywords: Prader-Willi Syndrome
ARD-101
PWS
Prader-Willi
Prader Willi
Prader Willi Syndrome
Lead sponsor: Aardvark Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-17
Enrollment: 90
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients
timeFrame: Baseline to Week 12
measure: Change in Clinical Global Impression of Severity (CGI-S) Score for Hyperphagia in Prader-Willi patients
timeFrame: Baseline to Week 12
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's of Alabama
state: Alabama
city: Encinitas
country: United States
facility: Rady Children's Hospital
state: California
city: Palo Alto
country: United States
facility: Stanford Childrens Health Specialty Services
state: California
city: Denver
country: United States
facility: Childrens Hospital Colorado
state: Colorado
city: Wilmington
country: United States
facility: Nemours Children Clinic Wilmington
state: Delaware
city: Gainesville
country: United States
facility: UF Shands Childrens Hospital
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Children's Hospital
state: Minnesota
city: Brooklyn
country: United States
facility: Maimonides Medical Center
state: New York
city: Mineola
country: United States
facility: NYU Langone Children's Ambulatory Care Center
state: New York
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Weslaco
country: United States
facility: Texas Valley Clinical Research, LLC
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: New Lambton
country: Australia
facility: John Hunter Children's Hospital
state: New South Wales
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Calgary
country: Canada
facility: Alberta Children's Hospital Research Institute
state: Alberta
city: Edmonton
country: Canada
facility: Li Ka Shing Centre for Health Research Innovation
state: Alberta
city: Montreal
country: Canada
facility: Centre Hospitalier Universitaire Sainte-Justine
state: Quebec
city: Toulouse
country: France
facility: CHU de Toulouse-Hôpital Des Enfants
state: Haute-Garonne
city: Angers
country: France
facility: CHU d'Angers
state: Maine-et-Loire
city: Paris
country: France
facility: AP-HP - Hôpital de la Pitié Salpétrière
city: Paris
country: France
facility: AP-HP - Hôpital universitaire Necker-Enfants malades
city: Toulouse
country: France
facility: CHU de Toulouse- Hôpital de Rangueil
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù IRCCS
state: Lazio
city: Milano
country: Italy
facility: Ospedale San Raffaele S.r.l. - PPDS
state: Lombardia
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
state: Toscana
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
state: South Korea
city: Incheon
country: Korea, Republic of
facility: Inha University Hospital
city: Suwon-si
country: Korea, Republic of
facility: Ajou University Hospital
city: Bucharest
country: Romania
facility: National University Hospital for Children's Neurorehabilitation "Dr. Nicolae Robanescu"
state: Bucuresti
city: Bucuresti
country: Romania
facility: Institutul National de Endocrinologie C. I. Parhon
city: Iaşi
country: Romania
facility: Spitalul Clinic de Urgenta Sfantul Spiridon
city: Sabadell
country: Spain
facility: Corporacio Sanitaria Parc Tauli
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron - PPDS
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
state: Leicestershire
city: Cambridge
country: United Kingdom
facility: Fulborn Hospital
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children (Glasgow) - PPDS - PIN
city: London
country: United Kingdom
facility: The Royal London Hospital
Study officials:
References: