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Due to operational and regulatory considerations.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARD-101 | Experimental | (Open-label) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARD-101 | Drug | 200 mg BID (twice per day) for 1 week, 400 mg BID for 1 week, 800 mg BID for 50 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | During treatment period (approximately 12 months) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's of Alabama | Birmingham | Alabama | 35233-1711 | United States | ||
| Children's Hospital of Orange Country |
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| During treatment period (approximately 12 months) |
| Orange |
| California |
| 92868 |
| United States |
| Stanford Children's Health Specialty Services | Palo Alto | California | 94304 | United States |
| Rady Children's Hospital | San Diego | California | 92123 | United States |
| Children's Hospital Colorado | Denver | Colorado | 80045-7106 | United States |
| Nemours Children's Clinic Wilmington | Wilmington | Delaware | 19803-3607 | United States |
| UF Shands Children's Hospital | Gainesville | Florida | 32610-3008 | United States |
| Emory University School of Medicine | Atlanta | Georgia | 30322-1047 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2991 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287-0005 | United States |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55102 | United States |
| Maimonides Medical Center | Brooklyn | New York | 11219-2918 | United States |
| NYU Langone Children's Ambulatory Care Center | Mineola | New York | 11501-4077 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232-0005 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | 2050 | Australia |
| The Children's Hospital at Westmead | Westmead | New South Wales | 2145 | Australia |
| Queensland Children's Hospital | South Brisbane | Queensland | 4101 | Australia |
| Alberta Children's Hospital Research Institute | Calgary | Alberta | T3B 6A8 | Canada |
| Stollery Children's Hospital | Edmonton | Alberta | Canada |
| Children's Hospital at London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| Inha University Hospital | Incheon | 22332 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Ajou University Hospital | Suwon | 16499 | South Korea |
| Fulborn Hospital | Cambridge | CB21 5EF | United Kingdom |
| Royal Hospital for Children (Glasgow) - PPDS - PIN | Glasgow | G51 4TF | United Kingdom |
| Leicester Royal Infirmary | Leicester | LE1 5WW | United Kingdom |
| The Royal London Hospital | London | E1 1BB | United Kingdom |
| ID | Term |
|---|---|
| D006963 | Hyperphagia |
| D011218 | Prader-Willi Syndrome |
| ID | Term |
|---|---|
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
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