FAMILIES
Rett Research Ready
Participating in clinical trials is a vital step toward discovering new treatments and, ultimately, a cure for Rett syndrome. Our Rett Research Ready™ program provides the education, resources, and support you need to make informed decisions about engaging in research opportunities.

Get Rett Research Ready
At the International Rett Syndrome Foundation (IRSF), we’re committed to helping you understand what it means to participate in a clinical trial and provide you with personalized research opportunities. We believe in a world without Rett. We invite you to join in the effort to bring new treatments — and ultimately a cure — to our loved ones.
What are the types of research?
Research is necessary to discover life-changing new treatments – and eventually a cure – for Rett syndrome. There’s Basic Research, Translational Research, Neuro-habilitation Research, and Clinical Research. Here, we define each type of research, and explain what each contributes to the goal of creating a cure.
Basic Research
Basic research investigates the life processes involved in health and disease. It’s usually performed in a laboratory using cells or animal models. This type of research may not apply directly to a treatment, but such investigations are essential to generate new ideas for therapeutic development down the line.
Translational Research
Translational research, also called “bench to bedside” research, tests something that came from basic research for its use in improving health outcomes. Like basic research, translational studies are typically performed in the lab using a model. The goal of translational studies is to produce a new treatment. Hopefully, the new treatment will prove safe and effective enough in an experimental setting to be tested in a clinical trial.
Neuro-habilitation Research
Neuro-habilitation research searches for ways to re-train the brain through cognitive, speech, occupational, and physical therapies. This type of research may involve animal studies or human participants. Rehabilitation professionals carefully supervise the research in a clinical setting. The data collected in neuro-habilitation research helps in the design of new treatment protocols that are based on science rather than isolated cases.
Clinical Research
Clinical research investigates health and disease in humans. One type of study that falls in this category is a clinical trial. This type of research requires human participants. It often takes place in a clinic setting, and a clinical team always oversees it. Surveys and neuro-habitational studies are other types of clinical research.
Clinical studies can be further broken down into two categories: Observational Trials and Interventional Trials. In an observational trial, researchers monitor participants’ health, collecting data on their disease symptoms and progression over time.
In an interventional trial, researchers study a participant’s response to a therapy. This therapy could be a drug, surgery, vitamin, exercise, or some other means of influencing disease processes. You can see the current list of interventional trials in myRett Trial Finder by searching by study type and selecting “Interventional.”
The time, money, and energy required of the families who choose to participate may vary by the individual clinical study. However, the goal of all clinical research is the same: to improve the quality of life for patients by advancing our understanding of a disease and its treatment.
How are new therapies developed?
The development of any new drug or therapy for Rett syndrome or any disorder must follow a scientifically rigorous and well-regulated path. This path is designed to make sure that any new therapy is safe and effective when it is made available to the public. The therapy development path is divided into five stages.
01
Discovery & Development
5-10 Years
02
Pre-Clinical Research
3-5 Years
03
Clinical Research
6-7 Years
04
Drug Review
1-2 Years
05
Post-Market Safety Monitoring
Months to Years
Safety & Dosage
Is It Safe for Humans?
A Phase I clinical trial tests a potential new treatment in people for the first time, sometimes called “first-in-human studies.” The primary goal of this testing is to determine if the treatment is safe in humans. This phase can also help identify a safe dose in humans.
Only a few volunteers (usually 10-100) are needed in this phase. The volunteers may be healthy “controls” (people without Rett syndrome) or may be individuals with Rett syndrome. A Phase I trial typically achieves its goal in months.
In addition to gathering information about the safety of a new treatment, a Phase I trial may also reveal:
- Any side effects
- The optimal treatment dose for humans
- The best way to give the treatment such as by mouth or injection
About 70% of treatment approaches tested in Phase I clinical trials advance to Phase II.
~70%
will advance to Phase 2
Safety & Efficacy
Is It Safe and Effective for the Target Disease?
The goal of a Phase II trial is to find out if the new therapy safely and effectively treats its target disease. These trials require more participants, and these participants must have the disease that the new therapy is intended to treat. Phase II trials also take longer than Phase I Clinical Trials to complete — usually months to years.
Sometimes Phase II trials assign participants into two groups by chance. One group receives the new experimental treatment. The other (the “control” group) receives either the standard of care intervention (whatever treatment is typically recommended for the disease) or a placebo (a substance that looks like the experimental treatment but is inactive).
Because no treatments currently exist for Rett syndrome, the “control” group in a Rett syndrome clinical trial would likely receive a placebo. Often, neither the participants nor the researchers know which treatment group an individual participant is in. This helps minimize bias in measuring a patient’s treatment outcomes.
If Phase II trials demonstrate that the new treatment is safe and effective at treating disease in the relatively small number of patients treated, the regulatory agency will allow Phase III testing to proceed.
Around 33% of new treatment approaches tested in Phase II trials meet the criteria to move on to Phase III.
~33%
will advance to Phase 3
Side Effects & Efficacy
Can Phase II Findings Be Confirmed?
Phase III trials aim to confirm or disprove the findings made in Phase II. They generally require more participants who have the disease the new therapy is intended to treat than Phase II trials do. That may be around 100 participants for a rare disease but can be thousands of participants for common conditions. This phase can often last several years and take place at several different medical centers.
Phase III trials usually randomly assign participants into either a “control” or “experimental” group. If a Phase III trial confirms the results of Phase I and Phase II trials for safety and efficacy, it can be considered for approval. During this approval process, regulatory agencies review the hundreds of pages of data collected on the new therapy. That review can take up to 12 months. Once approved, the new treatment is sent to pharmacies and may be prescribed to those who would benefit from it.
Approximately 25-30% of drug tests in Phase III move to regulatory approval. If approved, the new therapy can be brought to market and prescribed for use.
~27%
will advance to Phase 4
Continued Assessment
Does It Perform as Expected in the “Real World”?
Studies of a new treatment continue after it enters the market. The goal of these Phase IV clinical trials is to assess the new treatment’s performance in the “real world.” This is important because prior to its approval, the new treatment was only examined in a study setting. Many of the variables that are present in the lab or clinic are removed. These “real world” studies will reveal any long-term side effects and may detect any potential for the new therapy to treat other diseases.
How do clinical trials work?
Before any new treatment reaches the market, it must be tested in a series of interventional clinical trial phases to determine if it is safe and effective. Each phase must answer a different question about the new therapy. As such, each follows a different study design.
Phase 1
Safety & Dosage
Is It Safe for Humans?
Phase 2
Safety & Efficacy
Is It Safe and Effective for the Target Disease?
Phase 3
Side Effects & Efficacy
Can Phase II Findings Be Confirmed?
Phase 4
Continued Assessment
Does It Perform as Expected in the “Real World”?
Discovery & Development
5-10 Years
The development of a therapy begins in the lab as a basic research discovery. Scientists work for several months to years to generate these discoveries. In this stage, scientists identify new ways to modify a disease process or new disease-fighting technologies. They may identify a new drug, often by testing a library of 100,000+ compounds.
When they find a new treatment approach that looks promising in a first set of experiments, researchers gather more information to develop it. They get this information by performing experiments designed to answer questions about the new therapy, including:
- How does it react with the body?
- Are there any side effects?
- What is the best dose to use?
- Can the new treatment be modified to improve its potential to attain the desired outcomes?
- What is the best way to give the new treatment (such as by mouth or injection)?
- How does the new treatment compare to other treatment approaches to address patients’ health needs?
All these questions must be answered before a new treatment can be developed further. A large number of different experiments are required, which is why this stage of drug development is often one of the longest. Researchers perform all tests in this stage of drug development in cell culture or animal models. They review all the data they collect. This review gives researchers the information they need to determine whether or not to continue developing the new treatment.
Preclinical Research
3-5 Years
If a new therapy shows promising results in Stage 1, it moves on to the preclinical research stage (Stage 2). Preclinical research carefully tests the new drug or approach for potential to cause severe sickness, injury, or death in cell culture and animal models. This stage follows the regulatory agency mandated requirements. As in Stage 1, many different kinds of experiments are involved. The results of these experiments are carefully reviewed to determine if the new drug or approach is safe enough to test in humans.
Clinical Research
6-7 Years
If the sponsors leading the investigation of a new treatment or approach decide it looks safe and promising in preclinical testing and should be developed further, researchers design experiments to test it in humans. These clinical research designs define:
- Who qualifies as a participant?
- How many people will be involved?
- How long the study will last?
- How bias in the research will be limited?
- What data will be collected?
- How the data will be analyzed?
These trials are necessary to determine if the treatment produces the desired outcome in the human body. To protect human participants as much as possible, the new treatment sponsor must apply to the regulatory authorities for permission to conduct clinical research. In their application, the treatment sponsors must provide basic information about the new treatment (like how it is made), information about study investigators, the data obtained from preclinical testing, and all the details of their clinical research design.
A new treatment can only enter the clinical trial stage if regulatory authorities approve this application. The success of participant recruitment, the length of time participants need to be monitored, and how clear the results are all factors that affect the duration of the clinical research state.
Drug Review
1-2 Years
If the results from pre-clinical and clinical trials indicate that a new treatment approach is safe and effective, the sponsor can apply to the regulatory agency for permission to market it. During this drug review stage, regulatory authorities study all information collected about the new therapy. They then decide whether to approve, reject, or request additional data. If approved, the sponsor develops prescribing information with instructions for using the new treatment, and the treatment enters the market.
Post-Market Safety Monitoring
Months to Years after approval
Regulatory agencies continue to monitor the outcomes of patients being treated with a new therapy after its approval. As more people receive the new treatment, more data becomes available. This data may reveal new information about the therapy since it is impossible to find out everything during the therapy development process. Continued safety monitoring enables the prescribing information for a therapy to be changed if needed to protect patients.
Participating in Rett Research
Choosing to participate in a clinical trial is a big decision for your family. We want to help you understand your rights and protections as a research volunteer. Many regulations and safety measures are in place to ensure your loved one’s health and safety and minimize risks.
Your Rights & Protections
Clinical trials and the treatments that come out of them do not happen without volunteers. For rare diseases such as Rett syndrome, finding enough participants can be a significant barrier to a clinical trial’s success. Choosing to participate in a clinical trial is a big decision for your family. We want to help you understand your rights and protections as a research volunteer. Many regulations and safety measures are in place to ensure your loved one’s health and safety and minimize risks.
Although risks are always involved in clinical trial participation, regulatory authorities are responsible for making sure that these experiments are performed in the safest way possible. An Institutional Review Board (IRB), a group of doctors, scientists, and lay people who are not affiliated with the study, oversees many of these safety measures. The IRB reviews a study’s design and informed consent document. The informed consent is a form that all clinical trial participants must be given that outlines their rights, any risks involved in participation, and what to expect during the clinical trial.
The IRB must approve the study design and informed consent document before a clinical trial can begin. The IRB also monitors the study while it is ongoing to make sure the strict protocol detailed in the study’s design are followed. The group’s goal during the entire process is to protect the welfare of all study participants.
Before you join a study in any phase as a participant, you will meet with the researchers conducting the trial to discuss relevant aspects of the study and go over the informed consent form. Specifically, each study will vary in terms of who can participate, how many participants are needed, what tests will be used, how long the study will last, and how the data will be analyzed. It will also vary in terms of the time, money, and energy required of you and your family if you choose to participate.
You should ask as many questions of the researchers conducting the trial to get the information you need to decide whether participation is right for you and your family. Carefully consider what questions to ask before participating in a clinical trial.
Start the Conversation
Here are several questions to discuss with your Rett clinic team or clinical trial site as you consider enrolling in a clinical trial.
- What is the purpose of the clinical trial?
- Why do researchers think that this particular Rett syndrome treatment might work?
- Who is paying for and supporting the study?
- Who has reviewed and approved the study?
- Who will be in charge of my loved one’s care?
- How will the trial benefit my loved one or others?
- How long will the study last?
- How is the treatment given (e.g. orally, as an injection, etc.) and how often must it be taken?
- What will my responsibilities be if my loved one participates?
- What kinds of tests will be involved? How will data be collected (e.g. blood sample, behavior observation)?
- Do I need to stop any physical therapies or other interventions my loved one with Rett syndrome is receiving?
- What are the possible risks, side effects, and benefits?
- Can I talk to other people who are participating in the study?
- Will I be compensated for my participation in the clinical trial and my travel expenses?
- What is the time commitment for me? How many times will I have to visit the trial center? How long will each visit be?
- Will results of the clinical trial be made public and, if so, when?
- With whom should I communicate during the trial: the research team, my Rett syndrome care team, or both?
- When will I find out if my loved one received a placebo or the experimental treatment during the clinical trial?
Trial Conclusion
Most clinical trials proceed as designed from beginning to end, even when they don’t show the results the researchers hope for or expect. Completion of the study allows the scientific community to have conclusive results upon which to base their understanding and design future studies.
Clinical Trial Failures
Sometimes clinical trials fail despite the best efforts of everyone involved. Clinical trial failures are categorized as either a project failure or a research failure.
The most common reason for clinical trial failure is an inability to prove that a new treatment is effective. The reasons for not being able to demonstrate treatment effectiveness may include:
- The new therapy shows little or none of its disease-fighting effects researchers anticipated.
- The treatment may be effective, but:
- Researchers lack the tools or the data to show this effectiveness.
- The effectiveness of the therapy is smaller than anticipated and therefore not captured by the tool selected.
- The trial did not have enough volunteer participation.
Although not as frequent as failing to show efficacy, clinical trial sometimes fail due to safety issues or a lack of funding.
Continued Treatment After Trial Failures
Usually, volunteer participants can’t continue the treatment if the study fails. However, some exceptions can be made which depend on the treatment’s sponsor. The sponsor will consider several factors to decide whether or not to allow continued access to the treatment tested in the clinical trial, including:
- The new treatment shows a clear benefit for the individual taking it.
- The trial failure was due to efficacy or finances — NOT safety.
- The disease being treated is life-threatening.
- No alternative treatments are available.
If these conditions are met, and the sponsor is able to keep providing the treatment, the participant may be allowed to continue the new therapy. These exceptions fall under the compassionate use or expanded access programs allowed by regulatory bodies for unapproved therapies.
“One of the biggest takeaways for us participating in a clinical trial was that it gave us HOPE for the future, not only for Kayla, but for the whole Rett community. We don’t know what Kayla received in the trial (drug or placebo) but just the fact that she was receiving something that may help her and others down the line was a great source of hope for our whole family.”
Jenny Mosca
“Our family believes that each second of research is a leap towards a world where those who are diagnosed can live a life free from the barriers Rett syndrome presents. We hope that these studies help to give each child with Rett their best chance at living life to the fullest.”
Patty Mevis
“It was truly a rewarding, learning experience and we would do it all over if we knew it would help find a treatment. I encourage all parents to look carefully into the trial process, weigh all the factors of concern and move forward toward an experience of a lifetime.”
Jack Kuwik
Learn More
Frequently Asked Questions
How do I choose among the available studies?
This is an important and highly personalized question to consider. After reviewing relevant studies on myRett Trial Finder, it likely would be most helpful to talk with your loved one’s care team about which one(s) might be most appropriate.
Is there flexibility in when I come in for trial visits?
Every study or trial is different. Participation may last anywhere between weeks and years. Please refer to information about specific studies on myRett Trial Finder and talk with the team at your trial site.
What is the role of placebos in clinical research?
Rett syndrome is a complex disorder with lots of variability between individuals, but also within any one person with Rett on a day-to-day basis. For this reason, it can be difficult to assess improvement with the tools we currently have available. The placebo group helps to provide baseline variability against which a treatment effect is measured.
How is participating in research different than seeing a doctor?
While every researcher has the responsibility of safeguarding study participants, enrolling in clinical research is not the same as seeing your doctor for clinical care. Sometimes the researcher is also your regular Rett doctor, but here are some of the key differences to consider about what to expect during a clinical trial versus your regular medical appointments:
Research:
- The goal of research is to learn about your child’s illness.
- Research must use standardized procedures and protocols. In research studies, your child may be randomly assigned to a group taking a standard treatment or a placebo (an inactive substance or preparation used as a control) versus the group taking the treatment being studied.
- The safety and effectiveness of experimental medicines are not fully known, which is why they are being studied in a clinical trial.
- FDA-approved medicines are medicines that have already been studied in clinical trials previously and the side effects and benefits are better understood.
- Your child may be asked to undergo procedures (such as blood tests, EEGs, imaging scans, etc.) and you may be asked to complete questionnaires important for research but not necessary for your child’s care.
- Medical and other costs associated with participation in a research study may be partly or fully covered.
Clinical Care:
- The goal of clinical care is to treat your child’s condition.
- When providing clinical care, your doctor can change/customize your child’s treatment as needed.
- If necessary, a patient will always receive an active, FDA-approved drug.
- During routine clinical care of your child, testing procedures and questionnaires are restricted to those necessary for your child’s optimal medical care.
- You are responsible for treatment costs and/or the cost of health insurance coverage/co-pays associated with the routine medical care of your child.
What happens if I cannot attend clinical trial visits or do not want to continue?
As a clinical trial participant, you are considered a volunteer and you can withdraw your family member from the clinical trial at any point for any reason. There are many reasons one may withdraw from a study: difficulty making the protocol visits, difficulty tolerating the medication, difficulty remembering to give the medication. Withdrawing from a study does have a negative impact on the clinical trial as it adds cost and requires additional study participants to enroll to meet the number of study participants required for the study, but there are situations where it is in the best interest of the study participant to withdraw and that is okay. Some clinical trials will offer an open-label extension, meaning the study participant will be able to receive the study drug for a longer period of time even if he/she had received placebo during the study period, but in order to qualify for this open label extension one needs to complete the full placebo-controlled trial to be eligible. The open-label extension is a bonus for the research participants who went through the clinical trial. Open label studies (ones that do not have a placebo at all) are useful as small pilot studies to see if there is an effect of a therapy, but they are not sufficient for testing a drug to get FDA approval in most instances, which is why it is important to have placebo-controlled trials and to have participants willing to enroll.
What happens when the open label phase is complete?
If the study drug has not been approved by the FDA before the completion of the open-label phase, then an individual with Rett syndrome may be able to get the drug from their pharmacy with a prescription from their doctor. If the study drug has not been approved by the FDA before the completion of the open-label phase, then the study participant may not have access to the study drug any longer until the medication gets approved by the FDA.
Can I talk to someone who has been in a trial?
Yes. International Rett Syndrome Foundation is dedicated to helping you connect with information and resources as you consider clinical trial opportunities. This includes connection to clinical trial ambassadors. Please contact trials@rettsyndrome.org to ask about talking with a clinical trial ambassador.
How are decisions made about compassionate use of trial drugs after a trial concludes?
Compassionate use is a term used when an unapproved (experimental) medication is given to a patient not enrolled in a clinical trial. This is a decision that the sponsor (pharmaceutical company) makes and requires approval by the FDA to use the experimental medication under compassionate use.
How long does clinical trial participation typically last?
Every study or trial is different. Participation may last anywhere between weeks and years. Please refer to information about specific studies on myRett Trial Finder and talk with the team at your trial site.
Do I always have to go to the research site for clinical trial appointments? Can they ever be done from home?
Different clinical trials will have different types of visits and tests required. Some may allow for study visits to be completed from home (for example, with a visit from a nursing agency or going to your local lab for a test). Still, many require your study visits to be at the research site where the research volunteer enrolled in the clinical trial.
Why are there limited studies available in which boys can enroll?
Clinical trials are science experiments: testing to see if a drug works to improve a particular condition. Just like in a science experiment, the ideal clinical trial tests a similar population defined by the inclusion and exclusion criteria. The more variability in those that are enrolled in a clinical trial, the harder it may be to demonstrate that a study drug has benefit. For this reason, most clinical trials use classic Rett syndrome with a confirmed genetic mutation in MECP2 as one criterion required for enrollment. This will hopefully change in the future as our researchers learn more about males with Rett syndrome and can feel confident that the course of Rett syndrome in a male is similar enough to Rett syndrome in a female, therefore allowing males and females to both be included in clinical trials. The concern with including diverse population in a clinical trial is that it is too hard to demonstrate an effect, and therefore clinical trials are not successful, and the drugs cannot move forward in the process of FDA approval.
Is reimbursement for travel expenses available?
The short answer to this question is yes. There are various options available to ensure that travel expenses and logistics are not insurmountable barriers to participation in clinical trials and other research. Several sponsors offer travel reimbursement to families who travel for the purpose of participating in a clinical trial, and some sites provide options to help with logistics as well. Please contact International Rett Syndrome Foundation or Rettland for more information and support in accessing available resources.
Is it possible to switch trial enrollment mid-study if a different study feels like a better fit for my loved one?
It may be possible to discontinue enrollment in one trial and change to another. Clinical trial design and protocols will guide whether this is possible, as well as how long a participant would need to wait between trials. Please keep in mind that trials rely on participants to maintain their enrollment in order to meaningfully test the drug or therapy being tested or otherwise fulfill the objective(s) of the study.
Should we share details about a clinical trial on social media?
While a study participant and their family may want to share their experience with an investigational treatment, doing so may actually hurt the integrity of the study. For example, in sharing particular information (such as details of side effects), participants may discover who is on active study drug versus placebo, which can unblind the randomized controlled trial and lead to the discontinuation of a clinical trial. Not only would this create a loss for the pharmaceutical company, but all those hoping for approved treatments for their loved ones. For this reason, it is critical to remember that a clinical trial is an experiment to test if a therapeutic will work to treat Rett syndrome or some symptoms associated with Rett syndrome. These experiments require careful scientific rigor, and study participants should maintain that rigor to the best of their ability for the integrity of the study.
Which study holds most hope to cure Rett?
Rett syndrome is a severe and pervasive disorder. Trials aimed at symptom improvement or stopping the progression of the disease could significantly enhance one’s quality of life. These trials should be considered while we wait for studies that address the root cause of Rett syndrome, which are the most likely to offer potential transformative or curative responses. This research includes gene modifying therapies such as gene replacement therapy, gene editing, and RNA editing. Gene replacement therapy is the option that is closest to reaching clinical trials. The other types of gene modifying therapies are still being developed in the lab and are not yet ready for clinical trials in Rett syndrome.
¿Es un requisito que una persona hable inglés para inscribirse en un ensayo clínico?
Es un requisito que, por lo menos, un padre o cuidador(a) habla ingles para dar consentimiento informado para participar en el ensayo clínico y responder a preguntas de los investigadores. A veces el patrocinador de investigación clínica en los Estado Unidos busca la aprobación para realizar el ensayo clínico usando cuestionarios en español, pero es raro en un enfermedad rara como Rett.
Glossary of Terms
Adverse outcome/adverse event
Any undesirable experience associated with a patient’s use of a medical product.
Compassionate use/expanded access
When a patient with a life-threatening illness is granted access to an experimental treatment outside of clinical trials.
Crossover
A clinical trial method in which the participants receive two or more treatments (instead of just one) in a specific order.
Double Blind
A research method where neither the researchers conducting a clinical trial nor the volunteers participating in the trial know whether the participant is taking the experimental treatment or the placebo.
Efficacy
The performance of an intervention in a clinical trial.
Endpoint
An outcome that is being measured in a clinical trial.
- Primary– the main result or endpoint measured at the end of a clinical trial to determine whether or not a new experimental treatment works.
- Secondary– results that were obtained during a trial that can be analyzed, but are not necessarily controlled for.
Exclusion Criteria
Features that, if present, exclude an individual from clinical trial participation.
Habilitation
Habilitation refers to therapies to help an individual achieve or enhance skills that may have never developed, such as a child who is not talking as expected for their age. By comparison, rehabilitation helps people recover skills they once had but may have lost due to illness or injury. Individuals may experience enhanced quality of life from habilitation services at different ages and stages of their life to address functional abilities including mobility, communication, or activities of daily living.
Inclusion Criteria
Key characteristics that potential participants must have in order to be included in that trial, such as age range or disease progression.
Informed Consent
A process to inform potential study participants of the risks and benefits anticipated from a clinical study.
Institutional Review Board (IRB)
An administrative body consisting of doctors, scientists, and lay people that reviews plans for clinical research to protect the rights and welfare of the human subjects involved in that research.
Off Label
A use for an approved drug other than what that drug was designed for.
Open Label
A type of clinical trial where both the researchers and the participants know whether they are receiving the control or the experimental treatment. This type of study is the opposite of a double-blind clinical trial.
Open Label Extension
A continuation of a clinical trial in which all participants receive the study drug. Often, this type of study allows participants who were in a clinical trial to continue to receive the study drug for an extended period of time. Longer term safety data are collected in this type of trial.
Outcome Measures
Criteria used to assess the effectiveness or results of a clinical trial or the status of a disease or disorder in an observational study.
Phases
The steps of testing in a clinical trial that determine if a drug or therapy is safe and effective (see definition of Phase 1, Phase 2, Phase 3, and Phase 4).
Placebo
A substance that has no therapeutic effect that is often used as a control for testing new drugs. It is sometimes referred to as a “sugar pill.”
Placebo Controlled
A research method in which a substance that has no therapeutic effect (a placebo) is used as a baseline comparison (control) to measure the effectiveness of a new treatment.
Placebo Effect
A favorable change that occurs after taking a placebo (substance with no therapeutic effect). This phenomenon may be a result of hope or expectations of benefit, of increased visits to a medical provider, or to increased vigilance of daily activities. To test this effect versus a drug or therapy’s true benefits, clinical trials usually feature placebo-controlled designs.
Primary Outcome
The most important variable in answering a research question in a clinical trial.
Principal Investigator (PI)
The researcher that is leading the study.
Protocol
An outline of all the details about how a clinical trial is conducted, such as which tests should be used, how often tests should be performed, and what outcomes to measure.
Randomized Controlled Trial
A type of experiment that attempts to minimize bias in measuring the effectiveness of a new treatment. Participants are randomly assigned into two or more groups. One group (the “experimental” group) receives the intervention being studied. The other group (the “control” group) usually receives a placebo, an inactive substance that “looks” like the new treatment. When a treatment already exists, the control group receives the current standard of care.
Recruitment Status
Whether or not a trial is open for volunteer enrollment.
Registry
An organized system of uniform information used to evaluate specified outcomes for a predetermined clinical purpose.
Secondary Outcome
A variable that is of interest in answering a research question, but not as important as the primary outcome in a clinical trial,
Sponsor
A sponsor of a clinical trial is the entity, which could be a person, company, institution, group, or organization, that initiates a study and has responsibility to ensure protection of study volunteers and the collection of data that adheres to quality standards. Specific sponsor responsibilities include activities such as: oversight of the study, payment, selection of study sites, collection and analysis of data, and reporting and other communication with the Food and Drug Administration.
Therapy
An intervention that is intended to prevent, slow, stop or reverse a disease or symptoms of a disease. No therapy has been proven yet to change the course of Rett syndrome, but clinical trials and other research expand our understanding of Rett and offer hope for improved quality of life and treatment options.
Washout Period
In a crossover study, the time between treatments where a drug is washed out of a patient’s system before beginning the other treatment.
Our Sponsors
Programming made possible through the generous support of the following:



Rett Research & Clinical Pipeline
Stay informed about the latest developments in Rett syndrome research and the clinical pipeline. IRSF is your trusted partner in navigating the complexities of therapeutic development and clinical trials.