CLINICAL TRIALS FAQS

New treatments can only be developed with the support and partnership of the patient community.

Deciding whether or not to participate in a clinical trial can be a big decision, and potentially overwhelming. Understanding more about the clinical trial process and what to expect can help you make that decision.

What is a clinical trial?

A clinical trial is a research study conducted in people who participate as volunteers to test the safety, efficacy, dosing and more of a potential treatment. Regulatory authorities then use the results of clinical trials and other information to decide if a new medicine should be approved. There are strict rules for clinical trials, which are monitored by the U.S. Food and Drug Administration for trials conducted in the U.S. and other regulatory authorities for trials conducted in other countries.

What are the phases of clinical trials?
The clinical trial process is clearly outlined and monitored by regulatory authorities, such as the U.S. Food and Drug Administration. Clinical trials typically include four specific steps which help researchers collect different types of information about the treatment being studied. The steps are designated by four different phases, numbered 1-4, or sometimes using Roman numerals, I, II, III and IV.

  • Phase 1 – Testing in a small group of people, sometimes healthy volunteers, to better understand the treatment’s safety profile, including side effects, as well as information on the dose level.
  • Phase 2 – Testing with a larger group of people with a specific disease to find out if the drug is providing the desired effect and how effective it is. More safety information is also collected and multiple doses may be tested.
  • Phase 3 – Typically compares the effectiveness and safety of the new treatment against either placebo (inactive treatment or “sugar pill”) or the standard available treatment.
  • Phase 4 – After the treatment has been approved, an additional trial may be conducted to evaluate the safety and efficacy of a treatment over a long period of time. Phase 4 trials are not required for every treatment.

Sometimes you will see trials designated with more than one number, such as Phase 1/2. This indicates the trial is designed to gather data aligned with both steps of the clinical trial process.

Why should I participate in a clinical trial?
Developing new therapies takes years of research and clinical trials are a vital part of this process. Data gathered during a clinical trial is necessary to determine if a new drug or biologic is safe and effective in treating human diseases. Regulatory authorities then use the results of clinical trials and other information to decide if a new medicine should be approved. Volunteers who enroll in clinical trials help researchers understand the potential benefits and risks of these investigational therapies.

Deciding whether or not to participate in a clinical trial can be a big decision, and potentially overwhelming. While each trial is different, there are some common elements you can expect:

  • Clinical trial staff will walk potential participants through the informed consent process, which will include information about the study’s risks, logistics, and more. This information is to help the participant (and their family) make an informed decision if participating is the right option for them.
  • Trial lengths and the number of appointments and treatments provided will vary on what is being studied and the protocol of that specific trial. Some trials may only be a few weeks while others include long-term follow up that may continue for multiple years.
  • Depending on the trial protocol, overnight hospital stays may be required as well as participation in exams and test that are not part of the participant’s normal course of treatment.
  • Participants may be asked to report on their experiences in the trial by keeping a health log or journal, submitting forms, tracking with an app or other types of reporting.
How do I know if I am or my child is eligible? How do I enroll myself or my child in a trial?
Every clinical trial has a defined protocol for how the research will be conducted. The protocol includes eligibility criteria for participants. These criteria may include age, sex, disease severity, previous treatments, and more. There may be inclusion criteria – requirements you must meet – and exclusion criteria – conditions that would make you ineligible for the trial. Talking to your doctor is the best way to determine if a particular trial could be a good match for you or your loved one.

Once you and your doctor have identified an appropriate clinical trial, you may contact the study organizers. You typically can find contact information on clinicaltrials.gov or on the trial sponsor’s website. After you apply to participate in a trial, trial staff will use a screening process to determine if you are eligible and finalize participants. Not everyone who applies for a clinical trial will be selected to participate.

During the process, you will be provided with an informed contest document by the trial staff, which provides specific information about the trial protocol, study duration and requirements, and potential risks and benefits of participating. Before you sign the document, be sure to discuss anything that is unclear to you with the trial staff and/or your doctor. The informed consent document is not binding and you can leave the trial at any time for any reason.

What is informed content?
The process of communicating key information about a clinical trial to ensure that potential participants understand the risks and can make an educated decision about whether to participate. The information provided includes:

  • The study purpose
  • The treatment(s) given in the trial
  • Possible risks and benefits
  • Tests and exams required for the trial
  • Study duration

If you decided to participate in the trial, you will sign an informed consent form, but this is not a binding document. You can choose to leave the trial at any time for any reason.

Can I participate in a trial if my doctor is not a trial investigator?
Yes. It is common for participants to work with their regular doctor to enroll in a clinical trial. While in the trial, you will work with the study investigator and trial staff to receive your study treatment and undergo exams, but you will also maintain your relationship with your regular doctor.
How long do clinical trials last?
Trial lengths and the number of appointments and treatments provided will vary on what is being studied and the protocol of that specific trial. Some trials may only be a few weeks while others include long-term follow up that may continue for multiple years. The length and requirements for the trial will be shared with you in the informed consent process prior to enrolling in the trial.
Are trial participants paid? Do I have to pay to participate?
The clinical trial sponsor usually pays for all research-related costs and any special testing done as part of the trial. Depending on the specific trial, participants may be compensated for their time and/or reimbursed for travel-related costs. For information on a specific trial, you should review the informed consent document and/or speak to the trial staff.
If I enroll in a trial, can I change my mind and quit the trial?
Yes. You can leave a clinical trial at any time and for any reason.
Does location matter? What if I don’t live near a trial site?
Because each trial protocol has different requirements – for example, frequency of treatments, number of exams or overnight hospital stays – the necessity of being near to a trial site will vary. Many trials have study sites in multiple states and countries providing more trial site location options.

It may be possible to travel to a non-local site but the ability to attend the require treatments, medical procedures, etc. will depend on each participants’ individual situation. Your doctor and trial staff can help determine if a trial site’s location and travel requirements are suitable for you or your loved one.

Do trial medications always work?
No. The purpose of clinical trials is to gather data on the safety and effectiveness of a new treatment so regulators can determine if the treatment should be approved for use. Sometimes clinical trials show a treatment is not safe and/or effective for a group of patients or disease.

In addition, some clinical trials use a placebo (inactive treatment or “sugar pill”) which gives the researchers something to compare the new treatment too. Participants are randomly assigned to placebo or the new treatment and will not be told which they receive until after the “blinded” portion of the trial has been completed. Blinded studies are used to reduce bias and promote objectivity in the study results.

What is randomization? What does it mean that a trial is “blinded”?
When a study compares two or more treatments or a study treatment and a placebo, then patients are assigned to one treatment group by chance, not by choice, in a process called randomization. The participant will receive the highest level of professional care and expertise no matter which group they are assigned to.

In “double-blind studies” neither the participant nor the doctors and nurses know which treatment the participant is given, only the pharmacist knows. In “single-blind studies” only the patient is unaware of which treatment they are being given. Blinded studies are used to reduce bias and prevent anyone (doctors, nurses, or patients) from influencing the results. Double-blind studies are considered the gold-standard for trial designs and allow for scientifically accurate conclusions. If medically necessary, it is always possible for find out which treatment the patient is taking.

Will I get placebo? What happens if I get placebo instead of the trial medication?
Many studies are designed to compare a new treatment to placebo to determine the safety and efficacy of the treatment without bias. In trials like this, patients are assigned to one treatment group by chance, not by choice, in a process called randomization. The number of patients assigned to the placebo group vs the treatment group will vary based on the study protocol. The participant will receive the highest level of professional care and expertise no matter which group they are assigned to.

Some studies also offer participants the opportunity to stay in the trial for a long-term extension where patients who originally were assigned to the placebo group have the opportunity to receive the study treatment. The design of the trial, including use of a placebo and any long-term extension opportunities, will be included in the informed consent document.

Are there risks involved in participating in a trial?
Yes. While clinical trials are an important part of finding new treatments that can benefit patients, there are risks involved in participating in a clinical trial.

  • The treatment being studied may have side effects. Side effects can include anything from minor discomfort to potentially very serious ones.
  • The treatment being studied may not work for all participants or might not work equally for all participants.
  • In some trials, some participants may get a placebo (inactive treatment or “sugar pill”) rather than the treatment being studied.
  • The trial protocol may require more procedures, exams and/or time than the participant is used to, even the potential for overnight hospital stays.

The risks of participating in the trial will be explained during the informed consent process. A participant can leave the trial at any time if something occurs that causes them to change their mind about participating.

If I participate in a clinical trial, what protections are in place for me?
In the U.S., the Food and Drug Administration (FDA) is responsible for overseeing clinical trials and determining if the data resulting from clinical trials shows that a treatment is safe and effective for patients. Other countries have regulatory agencies that perform a similar role. Before a clinical trial can start studying a new treatment in the U.S., an Investigative New Drug Application must be submitted and cleared by the FDA.

The trial sponsor must develop and submit a trial protocol that is used by all the doctors participating in the trial. This protocol must be approved by Review Boards associated with the specific trial sites.

Informed consent is one of the many safeguards in place to ensure that patients have the information they need to balance the potential risks and benefits before agreeing to participate in a clinical trial.

How are trials measured for success?
All clinical trials have defined “endpoints” that are used to measure whether the trial met it’s objective or not. Endpoints may be a clinical endpoint, for example a functional assessment or extended lifespan, or a surrogate endpoint, for example a biochemical or genetic characteristic that can be objectively measured and are believed to be related to a clinical benefit.

A trial may include multiple clinical and surrogate assessments which means it may have more than one endpoint.

What happens when a trial ends – can participants continue getting the medication?
When a trial ends, the data collected is analyzed to assess the safety and efficacy of the new treatment. The data and other supporting material are provided to regulatory authorities, like the U.S. Food and Drug Administration, to determine appropriate next steps. Depending on the results and phase of clinical trial, the treatment may be submitted for approval or additional clinical trials may still be required.

Access to study treatment after the trial is completed varies. In some cases, trial participants can continue to receive the new treatment in a long-term extension of the trial. However, this is not always possible. The availability of a long-term extension may be covered in the informed consent document. If it is not, ask about this when considering whether to participate in a trial.

How do I contact someone with additional questions?

If you have questions related to Aeglea’s clinical trial, you can contact our clinical trial team.

If you wish to learn more about clinical trials, the FDA has developed a resource for What Patients Need to Know.

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