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) when used in a long term setting. It will also teach us about the safety of ARD-101. The main questions it aims to answer are: What medical problems do participants have when taking ARD-101 in a long term setting Does ARD-101 improve the total score of the HQCT-9 (hyperphagia questionnaire for clinical trials, 9 questions)? Eligible participants will: Have completed treatment on the AVK-101-301 study through Week 12/End of Treatment Take ARD-101 every day for up to 12 months. Visit the clinic at Months 1, 3, 6 and 12 during dosing and then have tele-visits at Week 2, Months 3 and 9, then 4 weeks after stopping the ARD-101. Patients/Caregivers will keep a daily diary.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
90
200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 50 weeks
Children's of Alabama
Birmingham, Alabama, United States
Children's Hospital of Orange Country
Orange, California, United States
Stanford Children's Health Specialty Services
Palo Alto, California, United States
Rady Children's Hospital
San Diego, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
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).
Time frame: During treatment period (approximately 12 months)
Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients
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.
Time frame: During treatment period (approximately 12 months)
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Nemours Children's Clinic Wilmington
Wilmington, Delaware, United States
UF Shands Children's 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
The Johns Hopkins Hospital
Baltimore, Maryland, United States
...and 20 more locations