How Patients Can Partner to Speed Treatments for PWS [WEBINAR]

On May 15, PWSA-USA and FPWR jointly presented the webinar How Patients Can Partner to Speed Treatments for PWS. The webinar provided important information about PWS clinical trials including: what to expect when participating in a trial, what questions you should ask, and how to get the information you need to decide if a clinical trial is right for your loved one with PWS. The webinar also gave a brief overview of the trial opportunities that are available in 2018. You can watch the webinar in its entirety or read a summary of the webinar below.


Webinar Summary

Half a dozen clinical trials are in phase two and three development. It is necessary to have volunteers from the community participate in these trials, because that is how we obtain data to determine the safety and efficacy of these drugs for treating PWS.

Drug development is a long process, beginning in preclinical work in the lab, then continuing to animal testing, followed by six to seven years of testing in human clinical trials. Phase two and three drug trials give longer-term evidence for safety and efficacy. Between 50 and 200 volunteers are needed for each phase two or three trial in order to get data we need to go on to the FDA for possible approval. 

We are getting closer to treatments, but we need to help the process by volunteering and supporting these clinical trials. One major reason that clinical trials are delayed or are not completed is due to an insufficient number of participants. The faster we can enroll the volunteers needed for these trials, the sooner we will have answers! 

This webinar will:

  • Provide more background on what is involved in participating in a clinical trial
  • Provide you with links and connections to other resources so you can learn more 
  • Summarize active or soon-to-be-active trials in case you wish to consider which trial to join

This webinar is intended to help you learn more about PWS clinical trials. The decision to participate in a trial is personal. Always consult your physician before making a decision. 

Benefits and Risks of Participating in Clinical Trials


Potential Benefits

Potential Risks
Immediate access to new, potentially beneficial treatment New drug may not work. Some participants receive a placebo.

Volunteers needed so that new treatments can be approved for the entire community

Risk of side effects

Even if the drug doesn’t work, the knowledge brings us closer to effective therapies

Participation can require significant time and effort
Insurance is not required; costs of medication, care and tests associated with the trial are covered There may be limits to reimbursement
Travel-related expenses are often covered

Travel time / energy / lost work usually not compensated

What to Expect When Participating in A Clinical Trial


Evaluation to determine if a volunteer should be included in the trial:

  • Demographics: Age
  • Particular characteristic: Genetic subtype, clinical features
  • Health: Potential examination to evaluate and rule out higher risk patients with other conditions (e.g. diabetes, high blood pressure)
  • Drugs currently being taken by participant: Concerns about interaction

Informed Consent

Legal definition: Voluntary agreement of an individual, or his or her authorized representative, who has the legal capacity to give consent, and who exercises free power of choice, without undue inducement or any other form of constraint or coercion to participate in research

Practical definition: Participant and/or guardian agrees to participate after gaining sufficient knowledge about the study and having all their questions answered such as:

    • Nature of the proposed research
    • Anticipated risks and potential benefits
    • Requirements of the research

Informed consent is an ongoing process.

Study Schedule

During the study there will be a required series of visits to the physician to perform an examination and to track a variety of health metrics used to evaluate the safety and efficacy of the drug.

      • Usually the drug (or placebo) is provided after the initial baseline visit
      • The visits tend to be frequent in the beginning and then slow down
      • During the study, health metrics will be collected:

        • Efficacy “endpoints”: e.g. weight, body composition, hyperphagia by questionnaires, behavior diary
        • Safety: e.g., blood chemistry, blood pressure, behavioral assessment by questionnaire
        • Pharmacology (PK/PD): e.g., blood draws at certain times after the drug is administered, urine testing
      • After the study, health metrics are evaluated statistically to determine if the drug was effective and safe.

Follow-Up / Reporting

  • Results of the study should be published in a medical journal 
  • Some studies will report results directly to you, some will not
  • In Phase 2/3 studies, often the Principal Investigator does not know ‘who got what’
  • Results reported on
  • Report to the community: webinar, meetings

Questions To Ask Before Participating In A Trial

Some basic information about clinical trials, including what to ask, can be found on the following websites:

Who should I talk to about participating in a trial?

  • Your doctor: The doctor or medical professional who follows your loved one with PWS closely is a good first person to have a discussion about participation in clinical trials. They may not have all of the details on any specific clinical trial, but can help you sort through possibilities.
  • Study coordinator at the site: This is usually the contact person on the clinical trial record. They will know all the logistics of the trial (eligibility, number of visits, procedures, etc).
  • Study investigator at the site: The doctor in charge of the trial at the clinical trial site will have all of the background information on the drug, provided by the sponsoring company. They should understand all of the known risks and benefits, and can advise you on whether the drug is potentially a good ‘fit’ for your loved one with PWS.
  • Institutional Review Board (IRB) Director: Each clinical trial site is overseen by an IRB director. If there are any concerns about the way the trial is being conducted, this person can help.

What Trial Opportunities Are Available?

There are multiple PWS clinical trial opportunities currently available for ages 3 through adulthood. Some trials are testing new drugs, others are intervention studies. Not all trials require travel, and several intervention studies can be participated in remotely. FPWR updates our information regarding trial opportunities regularly. You can view PWS clinical trial opportunities here.

Trial information is also available from the FDA at Information on this website is provided by the drug companies themselves.

You can also sign up for a monthly Clinical Trial Alert to get up to date information about new trial openings and new trial sites.

If you join the Global PWS Registry and click the button to be contacted for clinical trials, we will let you know when there is a trial that you might be eligible for, and provide you with contact information for the study.


Will a participant always receive the active drug or could he/she get a placebo?

All clinical trials are different. However, in many clinical trials, participants could receive placebo (a fake) either for all or part of a trial. This is done to provide a “control” to make sure that the effect of the active drug is real compared to the placebo.

In some trials with “parallel arms” (see glossary below), some patient will randomly be selected to receive placebo. In other trials (crossover trials) all patients receive both active drug and placebo bit they don’t know in which order.

Many studies have an “open label extension” where all trial participants (after an initial period when they might receive placebo) receive the active drug. If a trial has an open label extension, all participants will have the option to receive active drug during this phase.


Clinical Trials Glossary

Drugs in development go through three stages prior to approval, called Phase 1, Phase 2 and Phase 3.

  • Phase 1 is the first phase of in human tests and is usually done in healthy volunteers and is focused mainly on determining safety.
  • Phase 2 usually includes treatment in patients to judge efficacy and is often done to determine dosing. Safety is also monitored.
  • Phase 3 is the stage needing the most patients and lasts the longest. It is the typically done with randomized, placebo-controlled trials to establish safety and efficacy before FDA review.

Placebo—a “fake” drug that looks like the active drug but has no medicine. Placebo is given to compare results for an active drug to a fake drug to see if the active drug is actually making a difference.

Randomized— this refers to a trial that randomly chooses which patients are on active drug and which are on placebo.

Crossover study—is a study in which trial participants go thoruhg two phases of testing—one on active drug and one on placebo.  It is randomized whether one is put on active drug or on placebo first

Parallel arm study-- it is randomized whether one is put on placebo or on active drug

Blinded (single, double or triple)—to avoid bias, trials often prevent patients, doctors and evaluators from knowing who has received active drug or placebo. This is referred to as blinding or a blinded trial.

  • In a single blind study the patient does not know if he/she is receiving active drug or placebo.
  • In a double blind study, neither doctor nor patient knows who is getting drug or placebo.
  • With a triple blind trial study, neither the patient, the doctor or the person who evaluates response know who is getting active drug and who is getting placebo.

Open label study— an open label study is not blinded and there is no one taking placebo. Everyone knows that the patient is receiving the active drug.

Open label extension – in some clinical trials, there is a time after the randomized, blinded period in which all participants are allowed to receive the active drug if they choose.  This can be good for patients as it allows them to receive or continue an active drug, and it is good for the owner as they can gain more information about longer term safety and efficacy.  This period is known as “open label” since everyone knows the patient is on drug (ie, it is no longer blinded)

For updated information on PWS clinical trial opportunities and to sign up for a monthly PWS Clinical Trial Alert, visit our PWS Clinical Trials page.

PWS Clinical Trials


Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.