An Observational Study of Vykattm Xr (Diazoxide Choline Extended-Release Tablets) in Patients with Prader-Willi Syndrome (Pws)

Observational Study on Prader-Willi Syndrome Treatment

M
Melissa Lah, MD

Primary Investigator

Enrolling By Invitation
17 years or below
All
Phase N/A
5 participants needed
1 Location

Brief description of study

This is a prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with Prader Willi syndrome treated with VYKAT XR. The study will take place in the US. It is anticipated that approximately 200 patients will be enrolled and followed for up to 2 years in this observational study.

THIS STUDY IS ENROLLING BY INVITATION ONLY - Participants will be identified from the practice of the PI or her colleagues within IU Health. Patients with PWS at participating sites who are initiating treatment or have initiated treatment with VYKAT XR at the time of enrollment into this observational study, including patients who were previously treated with DCCR in a prior clinical study, will be invited to participate.  The study will be discussed with subjects/caregivers at routine clinic visits or via remote platform.

Detailed description of study

All patients in the study will receive care for PWS according to routine clinical practice in the respective enrolling site.

All patients in the study will receive care for PWS according to routine clinical practice in the
respective enrolling site. Clinical care will not be mandated by the protocol. The Sponsor will
not provide VYKAT XR or other treatments as part of this observational study protocol;
however, patients who receive VYKAT XR as part of a bridge supply or compassionate use
program will remain eligible to participate in this observational study.

All prospective treatment in this protocol will be commercial VYKAT XR prescribed to patients
by their physicians in addition to their standard of care; patients will not receive experimental
intervention or treatment from the Sponsor as part of their participation in this study.
Physicians are free to add or withdraw any medication and will continue to monitor the patient
for up to 2 years, or until withdrawal of consent, loss to follow-up, or death, whichever comes
first.

Patients will be followed prospectively for up to 2 years from the date of signed informed
consent (enrollment) in order to ascertain patient outcomes for this time period. If treatment with
VYKAT XR has been discontinued, patients may be followed for after discontinuation for up to
2 years total from the time of enrollment in this observational protocol. Follow-up will end with
withdrawal of consent, loss to follow-up, death, or end of study data collection, whichever comes
first.

No mandatory visits, tests, or clinical assessments are required for this study; however, patient
adherence assessments are important study components and highly encouraged. The patient
adherence assessments will be collected at least every 6 months after baseline, and at study
and/or treatment discontinuation.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Prader-Willi Syndrome, PWS, Riley
  • Age: 17 years or below
  • Gender: All

Inclusion criteria:

• Patients currently receiving or initiating treatment with
VYKAT XR, including patients who previously received
DCCR or are transitioning from DCCR to VYKAT XR in the
context of a prior DCCR clinical trial.
• Patients and/or caregivers must provide written informed
consent.
 

This study investigates Prader-Willi Syndrome (PWS), a genetic disorder that affects many parts of the body, often leading to a constant feeling of hunger and other symptoms. This is an observational study, meaning researchers will collect data by observing participants who are receiving routine care without changing their treatment. The focus is on patients using an investigational medication to understand its safety profile and outcomes better.

Participants in the study will receive their regular care for PWS, without any changes mandated by the study protocol. The investigational medication will be prescribed by their physicians as part of their standard care. There are no mandatory visits or tests, but adherence assessments are encouraged every 6 months to monitor how well patients follow their treatment plan.

  • Who can participate: Patients with Prader-Willi Syndrome currently receiving or starting treatment with the investigational medication, including those transitioning from a previous study, can participate. Written informed consent from patients or caregivers is required.
  • Study details: Participants will continue their regular treatment for PWS as prescribed by their doctors. The study does not provide the investigational medication, and no experimental treatments are given. Physicians can adjust medications as needed.
  • Study timelines: The study will last up to 2 years.
Updated on 09 Dec 2025. Study ID: MMGE-SOLENO-C623, 29390
Please visit our main page to search for other studies you may be interested in. If you need help finding a study or have any questions, please contact us at inhealth@iu.edu

Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only