Frequently Asked Research Questions 

Joining a research trial or study can be a wonderful way for contributing to our knowledge about disorders and their treatment. If you are interested, someone from the clinical trial staff will explain the details of the study, risks and benefits, and your rights as a participant, including your right to withdraw from the study at any point. Once all your questions have been answered, they will ask you to sign an informed consent form to be contacted for research and/or participate in a specific study.

Choosing to participate in a clinical trial or research study is an important personal decision. The following frequently asked questions (FAQ) provide detailed information about clinical trials and were modified from the NIH Clinical Trials website, the UCSF Human Subjects Protection Program website and the UCSF Memory and Aging website.

  1. What are the Autism Spectrum Disorders (ASD)
  2. What is clinical research?
  3. What is a clinical trial?
  4. Why participate in a clinical trial?
  5. What happens when a person signs up for a clinical trial?
  6. Who can participate in a clinical trial?
  7. What are some common inclusion criteria for clinical trials for ASD at UCSF?
  8. What are some common exclusion criteria for clinical trials for ASD at UCSF?
  9. What happens during a trial?
  10. What should people consider before participating in a clinical trial?
  11. What are other benefits and risks of participating in a clinical trial?
  12. Who pays for research?
  13. How is the safety of the participant protected?
  14. What should people ask before participating in a trial?
  15. Does a participant continue to work with a primary health care provider while in a trial?
  16. Can a participant leave a clinical trial after it has begun?
  17. Where do the ideas for trials come from?
  18. What is a placebo?
  19. What is a control or control group?
  20. What are the different types of clinical trials?
  21. What are the phases of clinical trials?
  22. What happens to my data?

What are the Autism Spectrum Disorders (ASD)?

Autism spectrum disorders (ASD) and pervasive developmental disorders are general terms for clinical brain disorders that affect a person’s ability to socialize and communicate. Individuals often also have narrowed or repetitive interests and behavior. Many individuals with also have difficulty with sensory processing, memory, attention, anxiety, organization, impulse control, and emotional regulation. The current diagnostic criteria were determined in the DSM 5.

What is clinical research?

Clinical research is generally considered to be a health-related research study in human beings that follow a predefined plan. The UCSF Sensory, Neurodevelopment and Autism Program supports both observational and interventional types of research.

  • Observational studies follow people who may already be receiving treatment for an illness while measuring their outcomes. These studies rely upon a more natural setting as participants select their treatment. This approach is often used when interventional studies would be unethical or impractical (such as in the case of a rare disease).
  • Interventional studies test promising treatments in a controlled environment. In these studies, the investigator assigns participants to particular treatment groups in order to measure and compare their outcomes at the end of the trial. These studies are also called "clinical trials" or "treatment trials." New treatments should not be widely used until they have been shown to work safely in patients, and clinical trials are the standard used to judge these treatments safe and effective.

What is a clinical trial?

A clinical trial is generally considered to be a biomedical or health-related research study in human beings that follow a predefined protocol. The UCSF Sensory, Neurodevelopment and Autism Program conducts both interventional and observational types of studies.

  • Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured. The information presented here will mainly pertain to interventional studies.
  • Observational studies are those in which individuals are observed and their outcomes are measured by the investigators.

Rapid advances in our knowledge about autism have led to the development of many new drugs and treatment strategies. However, before these new strategies can be adopted, they must be shown to work in humans. This means that clinical trials—studies to rigorously test how well a treatment works for patients; have become an increasingly important part of autism research. Advances in treatment are only possible through the participation of patients and study partners (a patient’s spouse, family member, caregiver or friend) in clinical trials.

Participating in a clinical trial is a big step for people with autism and their caregivers. That's why physicians and clinical trials staff spend time talking with participants about what it's like to be in a trial, and the pros and cons of participating. Here are some things that potential participants might want to know about clinical trials.

Why participate in a clinical trial?

Participants in clinical trials can play a more active role in their own healthcare, gain access to new research treatments before they are widely available, and help others by contributing to medical research.

What happens when a person signs up for a clinical trial?

Once someone has indicated (s)he is interested in participating in a clinical trial affiliated with the UCSF Sensory, Neurodevelopment and Autism Program, her/his name will be added to our list of interested potential participants. When trials become available, we make every effort to enroll all people who are interested in, and qualify for, a clinical trial in a timely fashion. Sometimes, due to the timing of a study, a cap on the number of people we can enroll, or other unforeseen circumstances, interested patients may have to wait until a spot opens up, or occasionally may not be able to participate at all. As soon as a patient indicates (s)he is interested in participating in a clinical trial, we will do our best to inform him/her about potential delays, limited enrollment or other time pressures that may affect participation.

It is important to learn about the study. We explain the trial in detail to potential research participants and describe possible risks and benefits. Staff also talk about the participants' rights as research volunteers, including their right to leave the study at any time. Participants and their study partners are entitled to have this information repeated and explained until they feel they understand the nature of the study and any potential risks.

Once all questions have been answered, and if there is still interest in being a part of the study, the patient is asked to sign an informed consent form. Laws and regulations regarding informed consent differ across states and research institutions, but all are intended to ensure that patient participants are protected and well cared for.

Age or mental capacity may prevent a patient from providing informed consent. In such cases, it is appropriate for an authorized representative (usually a parent) to give permission for the patient to participate. Next, patients go through a screening process (see inclusion/exclusion) to see if they qualify to participate in the study. If they qualify and can safely participate, they can proceed with the other parts of the study.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. The factors that allow someone to participate in a clinical trial are called inclusion criteria and those that disallow someone from participating are called exclusion criteria. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Using inclusion and exclusion criteria is an important principle of medical research that helps to produce reliable results. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. Before joining a clinical trial, a participant must qualify for the study. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

What are some common inclusion criteria for clinical trials for autism spectrum disorders at UCSF?

Here we list some examples of common general criteria for many of the studies that are done for ASD at UCSF. Each specific study will have its own set of criteria that may differ from those listed below.

  • Age in the range of 2–50 years old. Age ranges vary depending on the study focus (i.e., new onset, childhood, adult)
  • A clinical diagnosis of ASD. This diagnosis is usually made using parent history and direct observation of the study participant. The diagnosis will then be verified by a study clinician, usually at the first visit.
  • Cognitive status. Participants in clinical trials for ASD must have clear evidence of social and communication impairment, but must not be too impaired to perform the tests involved in the study. One common assessment that physicians use to measure cognitive status is called the Weschler Intelligence Scale for Children. The range for inclusion varies considerably from one study to another.
  • Caregiver Participation Having a study parent who has frequent contact with the participant and who is willing to come to the study visits. This criterion is part of most autism studies because most ASD patients may need assistance to drive or travel to study visits, remember appointments or reliably take their study medication(s). In addition, a caregiver’s opinion about how a study participant is doing is an important component of most studies.

What are some common exclusion criteria for clinical trials for ASD at UCSF?

  • Medications. Most current ASD clinical trials allow subjects to continue to take medications for other conditions. However, subjects are generally required to be on stable doses of these medications for a specified period prior to beginning a study. A stable dose means that there have been no changes in the drug dosage during that time period. Missing a dose or two will not affect eligibility.
  • Comorbid Condition. Common conditions that are considered exclusions for some clinical trials include: epilepsy and other psychiatric disorders. However, this will vary from trial to trial.

What happens during a trial?

If participants agree to join the study and the screening process shows they're a good match, they have a "baseline" visit with the study staff. This visit often involves cognitive and behavioral testing. This gives the study team information against which to measure changes during the study. Participants will then receive the test drug or treatment. As the study progresses, participating patients and family members must follow strict medication or treatment instructions and keep detailed records of symptoms. Participants may have periodic visits to the research center to have additional testing.

In most clinical trials, participants are randomly assigned to a study group. One group, the test group, receives the experimental drug or cognitive training. Other groups may receive a different drug or training or a placebo (an inactive substitute that looks like the study drug). Having the different groups is important because only by comparing them can researchers be confident that changes in the test group are the result of the experimental treatment and not some other factor.

In many trials, no one—not even the study team—knows who is getting the experimental intervention and who is getting the placebo or other intervention. This is called "masking," meaning that the patient/family member and the staff are "blind" to the treatment being received. Often, at the end of the trial, there is an opportunity for patients in the placebo group to take the experimental drug or participate in the active treatment protocol.

What should people consider before participating in a clinical trial?

Expectations and motivations. Clinical trials generally don't have miraculous results. The test drug or treatment may relieve a symptom, change a clinical measurement, or reduce the risk of death. With a complex disease like ASD, it is unlikely that a drug/or cognitive training will cure or prevent the disorder. Some people choose not to participate or drop out of a study because this reality doesn't meet their expectations. Others participate because they realize that even if the benefit to them may be slight, they are making a valuable contribution to knowledge that will help future patients.

Uncertainty. Some families have a hard time with the uncertainties of participation—not knowing whether the person is in the active group or the placebo group, not being able to choose which study group to be in, not knowing for a long time whether the study was successful or not. Ongoing and open communication with study staff can help to counter this frustration.

Finding the right clinical trial. Some clinical trials want participants who have good language ability or have only mild symptoms because they are testing a drug or training that might target higher order social skills. Other trials are interested in working with participants who have more severe ASD because they are testing a drug that might lessen behavioral symptoms, or they are testing new strategies to help caregivers. Even though a participant may not be eligible for one trial, another trial may be just right.

The biggest benefit of all. Many families find that the biggest benefit of participating in a clinical trial is the regular contact with the study team. These visits provide an opportunity to get state-of-the-art ASD care and also to talk on an ongoing basis with experts in ASD who have lots of practical experience and a broad perspective on the disease. The study team understands and can provide advice on the emotional and physical aspects of the person with ASD and the caregivers' experience. They can suggest ways to cope with the present and give insights into what to expect in the future. They also can share information about support groups and other helpful resources.

What are other benefits and risks of participating in a clinical trial?

Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

  • Play an active role in their own healthcare.
  • Gain access to new research treatments before they are widely available.
  • Obtain expert medical care at leading health care facilities during the trial.
  • Help others by contributing to medical research.

There are risks to clinical trials.

  • There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
  • The experimental treatment may not be effective for the participant.
  • The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

Who pays for research?

A clinical research study is paid for by the organization that sponsors the research. If the research is sponsored by the government, it may be paid for through a grant awarded to the doctors and scientists who conduct the research.

Some research studies are sponsored and paid for by drug and medical device companies. Other studies are paid for by private foundations for specific diseases, by gifts to the University or scientist, or by the researcher’s department at the medical center.

How is the safety of the participant protected?

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers may report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.

Before a clinical research study can begin, the research plan must be approved by an Institutional Review Board. An Institutional Review Board, also called an IRB, is a committee of doctors, scientists, and people from the community where the clinical research is taking place. An IRB reviews the clinical research plan to make sure people in the research study will be treated fairly and that any risks will be explained to them. The IRB at UCSF is called the Committee on Human Research (CHR).

What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the study team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the study team. Some of the answers to these questions are found in the informed consent document.

  • Who will be in charge of my care? Will (s)he profit from my participation in the study?
    • Each clinical trial will have a principal investigator who is in charge of the clinical trial and the care of the individual participants. Salaries are determined by the University of California and the principal investigator’s department are thus publicly available. A portion of some of the clinicians’ salaries may be paid by clinical trial funds, however the investigators do not directly profit from your enrollment.
  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
  • What kinds of tests and experimental treatments are involved?
  • How do the possible risks, side effects, and benefits in the study compare with my current treatment?
  • How might this trial affect my daily life?
  • How long will the trial last?
  • Will hospitalization be required?
  • Who will pay for the experimental treatment?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this study?
  • How will I know that the experimental treatment is working? Will results of the trials be provided to me?

Does a participant continue to work with a primary health care provider while in a trial?

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.

Where do the ideas for trials come from?

Ideas for clinical trials usually come from researchers but can also come from a public or private company. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials in humans. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

What is a placebo?

A placebo is an inactive pill, liquid, or powder that has no treatment value. In addition, cognitive training trails will often include a training arm with a protocol that is not thought to change brain function. In clinical trials, experimental treatments are often compared with placebos to assess the experimental treatment's effectiveness.

What is a control or control group?

A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

What are the different types of clinical trials?

  • Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
  • Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals or lifestyle changes.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.

What are the phases of clinical trials?

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:

  • In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20–80) for the first time to evaluate its safety, determine a safe dosage range and identify side effects.
  • In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100–300) to see if it is effective and to further evaluate its safety.
  • In Phase III trials, the experimental study drug or treatment is given to large groups of people (1000–3000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments and collect information that will allow the experimental drug or treatment to be used safely.
  • In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

What happens to my data?

Generally, a single person's data is combined with the data from many other people to form groups with similar characteristics. This group data is then analyzed so that conclusions or discoveries can be made and shared with the community.