Hyperphagia, Hyperphagia in Prader-Willi Syndrome, Prader-Willi Syndrome
Conditions
Keywords
Prader-Willi Syndrome, ARD-101, PWS, Prader-Willi, Prader Willi, Prader Willi Syndrome
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) 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.
Interventions
DRUGARD-101
200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 50 weeks
Sponsors
Aardvark Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
13 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients who have completed treatment on Aardvark Therapeutics clinical study AVK-101-301
Exclusion criteria
* Any complications that makes participation unsafe in the Investigator's opinion
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score | During treatment period (approximately 12 months) | 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
| Measure | Time frame | Description |
|---|---|---|
| Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients | During treatment period (approximately 12 months) | 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. |
Countries
Australia, Canada, South Korea, United Kingdom, United States
Outcome results
None listed