It is an exciting time in PWS research, with multiple new clinical trials on the horizon this spring and through the end of 2018! There is already buzz throughout the community about upcoming studies, providing a wonderful opportunity for an open dialogue about the clinical trial process. FPWR does not advocate certain trials over others. We aim to provide resources for information, so that families are empowered with the knowledge and education to make the best decisions for themselves.
FPWR maintains a list of PWS clinical trials opportunities available (you can subscribe to email updates on that page as well), and we also publish details on specific clinical trials. This includes very high level information such as the study goals, age ranges of participants, and locations of study sites. When available, weblinks are provided for each study, linking directly to the study on the clinicaltrials.gov site. This site includes in depth information including detailed “Eligibility” inclusion/exclusion criteria, as well as contact information for the coordinator of the study. This person will be knowledgeable about the trial and will be able to answer all of your questions. Please remember that contacting a study coordinator to ask questions is not an obligation to participate in the study.
Learning about eligibility for a trial
Inclusion/exclusion criteria for each individual trial may include such things as: age, BMI, medication use, chronic illnesses, type of PWS, etc. These criteria are very carefully designed with the intention of maximizing benefit and minimizing risk, and are scientifically based upon the pre-clinical and early clinical data for that specific therapy. So, for example, if a drug is being evaluated for effectiveness in weight loss, it’s likely the participants will need to have a BMI in the “obese” range, so that the effect of the drug can be readily measured. Other drugs may not have weight loss as a primary measure, and so may allow lean or modestly overweight participants. Most trials do require a genetic confirmation of PWS, but will vary in terms of level of cognitive function or communication required. Participants are not able to be enrolled in more than one trial at a time. This would make it too difficult to tease out the benefits and or risks/side effects of each drug.
However, it is possible to complete a trial and to then participate in another, later trial. This is often addressed in the inclusion/exclusion criteria wherein there may be a requirement to have not participated in any other trials over a certain time frame, e.g. the past 6 months. Each trial, even if with the same drug, is completely independent with regards to seeking participants (e.g. participants from a phase 2 trial of specific drug are not automatically enrolled in phase 3 of the same drug). Inclusion/exclusion criteria can change through phases of trials, based upon previous results and/or the goals of the current study. There are usually no limitations on where you live, as long as you are able to make the required travel/visits/stays.
Deciding whether or not to participate in a trial
If your family meets the inclusion/exclusion criteria for a trial, or is fortunate to be eligible for more than one trial, you will want to start gathering information on the trial(s) and asking a lot of questions. It is important to understand that seeking information is in no way a commitment to participate. It is an opportunity to learn about the details of the study and find the facts you need to make the best decision for your family. In addition, you are able to withdraw from any study at any time. Your points of contact during this process will be the study coordinator and principal investigator (PI) for each individual trial.
It is important to get to know and be comfortable with the investigators of a study, to develop a relationship of trust where you know all of your questions and concerns are fully addressed. Some of the types of questions that should be directed to the study coordinator may include: trial length, number of visits, length of visits, financial assistance for travel, as well as adverse events and potential side effects.
Clinical trials are research studies
It is important to remember that a clinical trial is a research study. Basically, there is a scientific reason/rationale to support why the drug or therapy may work, but the only way to know if it is actually going to work in patients is to conduct the trial. The best way to demonstrate a true effect of the drug is to compare it to a placebo, in fact, in most cases, the FDA will only approve a drug if it has been studied in a blinded, placebo-controlled clinical trial. This is the most rigorous study design and is the least likely to give false results, and therefore the most widely used to gain FDA approval. Drug companies and researchers do recognize that it may be frustrating for families to participate in a clinical study and never receive the drug.
To address this, may trials offer a crossover design where where participants receive drug for part of the trial and placebo for part of the trial. Some studies also include an “open label extension”, which allows everyone at the end of the study to choose whether or not they would like to receive the drug. This part of the study is not blinded and the data gathered is not as robust as the blinded, placebo-controlled portion of the trial, but it can be beneficial to get more data, and beneficial for individual participants to see if the drug has a beneficial effect for them.
Clinical trials are categorized into “Phases”
When reading about clinical trials, you will see them described as Phase I, II, III, or IV. These are basically steps along the drug development process. A drug will progress through Phases 1-III before reaching the review and approval stage by the FDA. The earliest phase is a small group, focused primarily on dose, safety, and side effects. Phases II and III are progressively larger and focus on efficacy. For detailed information about the drug development process and the differences between the various Phases of clinical trials, please click here for a video of FPWR Director of Research Programs, Theresa Strong PhD.
What happens after a clinical trial is over?
In general, once a clinical trial is completed, participants will not have access to the drug again until either they are able to enroll in a next phase trial, or the drug is approved and comes to market. Until a drug approved, the only way to gain access to it is through a clinical trial. It is important to remember that the clinical trial process is at the core, a careful scientific experiment. All data must be collected, compiled, and analyzed in order to determine how to proceed.
Stay up to date on PWS clinical trials!
For an overview of PWS clinical trials currently available or opening soon, click here.