This study is NOT currently recruiting participants.
Number
001723-CH
Sponsoring Institute
National Institute of Child Health and Human Development (NICHD)
Recruitment Detail
Type: No longer recruiting/follow-up only Gender: Male & Female Min Age: 6 Years Max Age: 100 Years
Referral Letter Required
Yes
Population Exclusion(s)
Pregnant Women
Keywords
Prader-Willi Syndrome; Hyperphagia; Diazoxide; Weight Loss
Recruitment Keyword(s)
None
Condition(s)
Prader-Willi Syndrome
Investigational Drug(s)
DCCR
Investigational Device(s)
Intervention(s)
Drug: DCCR
Supporting Site
National Institute of Child Health and Human Development
Prader-Willi syndrome (PWS) is a complex genetic disorder. It can cause problems including uncontrollable hunger, short stature, and decreased muscle tone. Better treatments are needed.
Objective:
To study the long-term effects of diazoxide choline (DCCR) extended-release tablets in people with PWS.
Eligibility:
People with PWS who participated in protocol 19-CH-0026 at the NIH Clinical Center.
Design:
Participants will be in this study for up to 5 years.
Participants will have yearly visits to the NIH clinic. They will have a physical exam with blood and urine tests. They will have tests of their heart function. The study doctor will press a finger onto the participant s foot, ankle, and shin to check for excess fluid under the skin (edema). Participants will be checked for an unusual amount of hair and signs of blood clots.
DCCR is a tablet taken by mouth. Participants will be given a supply of DCCR to take at home. They will be given a diary; they will write down when they take each dose. They will write down any adverse effects they may have.
Other study visits can be by telephone or telemedicine. These visits will be every 2 weeks for the first 6 weeks of the study, with 3 more in the first year. Then there will be 2 visits per year.
In the morning before each call, participants will prick their finger to draw blood to test their blood sugar at home. They will talk about their symptoms and check for edema and blood clots. For some visits, participants may to go a local clinic for blood and urine tests.
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INCLUSION CRITERIA: Potential participants must meet all of the following inclusion criteria to be enrolled: -Provide voluntary, written informed consent (parent(s) / legal guardian(s) of the participant as appropriate); provide voluntary, written assent (participant as needed). -Participant must be willing to have telemedicine visits at 3, 6 and 12 months after Visit 1 then at 12-month intervals thereafter. -Participant must: --Have participated in and completed the Study C602 RW Period; --Have participated in but discontinued from the Study C602 RW Period and at least 16 weeks have elapsed since the date of their randomization into the C602 RW Period; or --Have participated in Study C602 OLE period, did not consent to participate in the RW Period, and at least 16 weeks have elapsed since the date of their C602 OLE End of Treatment Visit. EXCLUSION CRITERIA: Eligible participants must not meet any of the following exclusion criteria to be enrolled: -Positive urine pregnancy test (in females of child-bearing potential). -Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation. -Anticipated requirement for use at any time during the study of the following prohibited medications: --Medications known to prolong the QTc interval (Refer to QT Drugs List on https://crediblemeds.org/healthcare-providers/), except citalopram or escitalopram --Medications, including homeopathy and herbal preparations, that are strong inhibitors of CYP450 1A2 (Refer to https://drug-interactions.medicine.iu.edu/MainTable.aspx, Drug Interactions Flockhart Table (Trademark)) --Systemic steroids (i.e., oral, IM, or IV) for > 7 days -Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 OLE EOT Visit.
Potential participants must meet all of the following inclusion criteria to be enrolled:
-Provide voluntary, written informed consent (parent(s) / legal guardian(s) of the participant as appropriate); provide voluntary, written assent (participant as needed).
-Participant must be willing to have telemedicine visits at 3, 6 and 12 months after Visit 1 then at 12-month intervals thereafter.
-Participant must:
--Have participated in and completed the Study C602 RW Period;
--Have participated in but discontinued from the Study C602 RW Period and at least 16 weeks have elapsed since the date of their randomization into the C602 RW Period; or
--Have participated in Study C602 OLE period, did not consent to participate in the RW Period, and at least 16 weeks have elapsed since the date of their C602 OLE End of Treatment Visit.
EXCLUSION CRITERIA:
Eligible participants must not meet any of the following exclusion criteria to be enrolled:
-Positive urine pregnancy test (in females of child-bearing potential).
-Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation.
-Anticipated requirement for use at any time during the study of the following prohibited medications:
--Medications known to prolong the QTc interval (Refer to QT Drugs List on https://crediblemeds.org/healthcare-providers/), except citalopram or escitalopram
--Medications, including homeopathy and herbal preparations, that are strong inhibitors of CYP450 1A2 (Refer to https://drug-interactions.medicine.iu.edu/MainTable.aspx, Drug Interactions Flockhart Table (Trademark))
--Systemic steroids (i.e., oral, IM, or IV) for > 7 days
-Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 OLE EOT Visit.
Principal Investigator
Referral Contact
For more information: