Hyperphagia, 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). 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.
Interventions
DRUGPlacebo
200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 10 weeks
DRUGARD-101
200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 10 weeks
Sponsors
Aardvark Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
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 * At least 7 years of age or older in the US at the time of consent * At least 10 years of age or older in Australia * At least 13 years of age or older in countries outside of the US and Australia
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 any commercially available medication for the treatment of hyperphagia (i.e., Vykat) within 60 days of randomization and throughout the study. * Very high doses of glucocorticoids in the previous 3 months of Visit 1 and throughout the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Australia, Canada, South Korea, United Kingdom, United States
Outcome results
None listed