WUH: A randomized double-blind placebo-controlled study of diazoxide choline controlled-relealese tablet (DCCR) in patients with prader-Willi Syndrome.
Are you eligible to participate in this study?
You may be eligible for this study if you meet the following criteria:
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Conditions: Prader-willi Syndrome
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Age: Between 4 years - 80 years
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Gender: Male or Female
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Other Inclusion Criteria:
- Subjects who are at least 4 years old
- Subjects who will provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient) and provide voluntary, written assent (patients, as appropriate)
- Subjects with genetically-confirmed Prader-Willi syndrome by methylation analysis
- Subjects who scored 13 or greater on the Hyperphagia Questionnaire for Clinical Trials
- Subjects who are in stable care setting for at least 6 months
- Caregiver must have been caring for the patient for at least 6 months prior to Visit 1 and will care for the patient throughout the study a minimum of 4 waking hours per day
- Subjects and caregiver agree to continue the patient on his/her current dietary and physical activity regimens throughout the study
- Subjects with a diagnosis of type II diabetes mellitus are adequately managed with anti-diabetic medication(s) other than insulin and no recent history of ketoacidosis or hyperosmolar coma
- Subjects on stable regimens of all concomitant chronic medications for at least 3 months prior to Visit 1, except what is listed in the protocol
- Subjects who are able to complete all screening assessments and procedures
You may not be eligible for this study if the following are true:
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- Subjects with weight < 30 kg or =135 kg
- Subjects have participated in an interventional clinical study
- Subjects with History of allergic reaction or significant intolerance to Diazoxide or Thiazides or Sulfonamides
- Subjects who may use within 3 months prior to Visit 1 or anticipated requirement for use at any time during the
study of the following prohibited medications:
- Anti-obesity medications or other medications (including herbal preparations, over-the-counter products) or procedures for weight reduction
- Medications, including homeopathy and herbal preparations, that are strong inhibitors or inducers of CYP450 1A2 or 3A4
- Medications known to prolong the QTc interval
- Systemic steroids
- Any drugs medications, herbal preparation, homeopathy, nutraceuticals, or procedures (i.e., acupuncture, vagal stimulation), that may have an effect on any study endpoints
- Use of any investigational drugs or devices
- Subjects who anticipate changes in caregiver, care setting or other similarly potentially disruptive changes during participation in the study
- Subjects with known type 1 diabetes mellitus
- Subjects with uncontrolled hypertension
- Subjects with history of thromboembolic events, concurrent treatment for thromboembolic event(s), and/or signs suggestive of a possible thromboembolic event
- Subjects with known history of thromboembolic events in a first degree relative
- Subjects with Grade 3 or 4 peripheral edema at physical examination or history of severe peripheral edema that could not be managed with an oral diuretic
- Subjects who are pregnant or breastfeeding or with positive urine pregnancy test
- Subjects with history of torsades de pointes (TdP), congenital long QT syndrome, brady arrythmias, or uncompensated heart failure
- Subjects with diagnosis of psychiatric disease or hospitalization for a psychiatric reason within 6 months prior to Visit 1
- Subjects with cancer within the past 5 years
- Subjects with any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol
- Subjects whose primary caregiver cannot read and understand English, or communicate with Investigator and study site personnel
If you are registered as a volunteer, please log in to contact the study team/express interest in this study.