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
ENROLLING_BY_INVITATIONStanford Children's Health Specialty Services
Palo Alto, California, United States
ENROLLING_BY_INVITATIONRady Children's Hospital
San Diego, California, United States
ENROLLING_BY_INVITATIONChildren's Hospital Colorado
Denver, Colorado, United States
ENROLLING_BY_INVITATIONChange 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)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Nemours Children's Clinic Wilmington
Wilmington, Delaware, United States
NOT_YET_RECRUITINGUF Shands Children's Hospital
Gainesville, Florida, United States
NOT_YET_RECRUITINGEmory University School of Medicine
Atlanta, Georgia, United States
ENROLLING_BY_INVITATIONAnn and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGThe Johns Hopkins Hospital
Baltimore, Maryland, United States
NOT_YET_RECRUITINGUniversity of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
NOT_YET_RECRUITING...and 32 more locations