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Help us find new treatments for Prader-Willi syndrome (PWS) today

The CARE-PWS clinical study for 7 to 18 year olds with PWS is enrolling now


About Prader-Willi syndrome

Prader-Willi syndrome (PWS) is a rare genetic disorder. In people with PWS, the part of the brain that controls feelings of fullness and hunger does not work properly.

People with PWS may experience symptoms such as:

  • A constant feeling of hunger, known as hyperphagia

  • Obsessive and compulsive behaviors

  • Anxiety and mood disorders

These are not all the symptoms, and your loved one may experience different ones.

About CARE-PWS

If you have a loved one with Prader-Willi syndrome (PWS), he or she may qualify to participate in the CARE-PWS clinical study. The goal of CARE-PWS is to see if an investigational medicine is safe and effective at reducing PWS symptoms. There is no baseline weight or body mass index (BMI) requirement for people participating in this clinical study.

The investigational medicine will be administered through a nasal spray. Caregivers will have to observe and document their loved one’s experiences during their participation in the clinical study. This clinical study is enrolling participants who are:

  • 7 to 18 years old

  • Diagnosed with PWS

There is no cost to participate in the study, and reimbursement for study-related travel expenses may be provided.


Clinical Study Sites

The following sites are enrolling participants in the CARE-PWS clinical study. 

 

Take the next step to learn more about the CARE-PWS study by completing this form.


Frequently Asked Questions

WHAT IS INVOLVED IN THE CARE-PWS CLINICAL STUDY?

This clinical study is enrolling 7 to 18 year olds with Prader-Willi syndrome (PWS). If your loved one has been diagnosed with PWS, he or she might be eligible to participate in the clinical study.

Before enrolling in the clinical study, you and your loved one will need to understand what is involved by talking to the clinical study staff and reading the Informed Consent Form. The Informed Consent Form outlines what is involved in the clinical study as well as all the known safety information about this investigational medicine.

Once you sign the Informed Consent Form and visit the clinical study site, you and your loved one will visit your CARE-PWS study doctor for certain tests to make sure he or she is eligible to participate in the study. The clinical study doctor and study team will be available to answer any questions that you and your loved one may have as you go through this process.


WILL MY LOVED ONE RECEIVE THE INVESTIGATIONAL MEDICINE?

Everyone in this clinical study will receive either the investigational medicine or a placebo for the first 8 weeks of the study. After the initial 8-week period, all participants will receive the investigational medicine for up to 56 weeks.

Neither you, your loved one, your clinical study doctor, nor the clinical study staff will know whether your loved one is taking the investigational medicine or the placebo during the initial 8-week period. A computer program randomly chooses which nasal spray he or she will get, like tossing a coin. The results are then compared at the end of the clinical study.

WHAT SIDE EFFECTS SHOULD WE EXPECT?

The Informed Consent Form will review in detail all of the safety information known about the investigational medicine. After you read it, please direct any questions to your clinical study doctor.

DO WE HAVE TO PAY TO PARTICIPATE IN THE CLINICAL STUDY?

No. The investigational medicine and associated procedures/testing are provided at no cost to clinical study participants.

DOES THIS CLINICAL STUDY OFFER TRAVEL REIMBURSEMENT?

Participants in this clinical study may qualify for reimbursement for study-related travel.  

HOW DO WE GET STARTED?
Click below to complete the form to see if your loved one is eligible.


About Clinical Studies

WHAT IS A CLINICAL STUDY?

A clinical study is designed to understand whether an investigational treatment is safe and effective for people. There are usually several phases of a study, each designed to learn more about the investigational treatment’s effects. Clinical studies are divided into 3 phases:

  • Phase 1 is designed to gather information on safety and dosage

  • Phase 2 focuses on how well the medicine works

  • Phase 3 evaluates how well the medicine works and whether it is safe to take. The FDA uses this information during its consideration of approving the medication 

All clinical studies must follow a strict set of rules that are outlined in the study’s protocol. These rules set specific requirements on which types of patients can be included in the study, procedures that must be followed, and the number of study visits required.

Because of these rules, it is important that participants carefully follow the visit schedule and instructions from the study staff. Clinical studies are required for all new treatments in order to show that the treatment is safe and effective before it can be made available to the general population.

WHY ARE CLINICAL STUDIES CONDUCTED?
Clinical studies are conducted to answer research questions and to learn if a certain investigational medicine is safe and effective. The main questions researchers want to answer are:

  • Does this investigational medicine work?

  • What, if any, side effects does this investigational medicine cause?

  • Do the benefits of the investigational medicine outweigh the risks?

  • Which patients are most likely to find this investigational medicine helpful?

WHY SHOULD I PARTICIPATE IN A CLINICAL STUDY?

Participating in a clinical study is voluntary, and it is always up to you to decide if you’d like to do so. Reasons people participate in clinical studies include the following:

  • There is no current approved treatment for their condition or symptoms

  • They have not experienced improvement of their condition after using the currently approved treatments

  • They want access to a new type of treatment even if it is not guaranteed to be better or as good as already-available treatments

  • They want to contribute to the development of a new treatment for others with a similar disease