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

Phase 3

Recruiting

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.

Recruitment & Eligibility

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.
Very high doses of glucocorticoids in the previous 3 months of Visit 1 and throughout the study.

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.

Locations (44): Children's of Alabama
🇺🇸
Birmingham, Alabama, United States
Rady Children's Hospital
🇺🇸
Encinitas, California, United States
Children's Hospital of Orange Country
🇺🇸
Orange, 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
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸
Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸
Boston, Massachusetts, United States
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Children's of Alabama
🇺🇸Birmingham, Alabama, United States
Hussein Abdul-Latif, MD
Contact
Tushima Reeves
Contact
205-638-9173
tfails@uabmc.edu