Study

What is the purpose of the Prader-Willi syndrome study?

This is a Phase II study looking at the safety, tolerability, and efficacy of NNZ-2591 in the treatment of children with Prader-Willi syndrome.

This is an open-label study which means that every participant (and caregiver) will know they are receiving active treatment.

This study investigates an oral medication called “NNZ-2591”, which aims to improve the impaired connections and signaling between brain cells that are involved in Prader-Willi syndrome.

The study medication is experimental.

What does the study involve?

Participation in the study will be required for approximately 21 weeks, with a combination of in-clinic, remote, and telehealth consultations. An in-home nurse will also visit your house to check on the participant and conduct a number of assessments.

To ease the burden of travel on families, an in-home nurse will be available to assist with telehealth/remote visits. At a time convenient to yourselves, the in-home nurse will visit your home and conduct a number of the required study assessments. This has been included in the study design to make it easier for your family to participate in the study and promote inclusion, without compromising on the data that needs to be collected at each visit.

Participants in the Prader-Willi study will need to:

  • Attend clinic appointments
  • Take study medication (NNZ-2591)
  • Have blood, urine, and stool samples collected for laboratory testing
  • Have Prader-Willi syndrome symptoms monitored
  • Have heart activity monitored
  • Caregivers will also need to complete a number of assessments capturing their view of the participant’s status.

Is there a cost?

There is no cost to participate in this clinical trial.

Reimbursement is available for reasonable travel, lodging, and incidental costs incurred attending your clinic visits.

More details on this can be found by clicking through to our Resources page here

Who is eligible for this study?

Potential participants must:

  • Have a diagnosis of Prader-Willi syndrome.
  • Be aged between 4-12 years old.
  • Be able to swallow a liquid medication.

Specific details on the eligibility criteria can be found at www.clinicaltrials.gov

The clinical team managing the study at one of the participating sites will determine if your child is eligible to enroll. For more information, please speak with one of the participating study teams.