A-T is a rare, neurodegenerative, genetic disease with no approved therapies currently available for its neurological symptoms. The severity of A-T symptoms varies between individuals and at different ages. The most common symptoms of A-T include:
Previous studies have suggested that drugs like the study drug may help patients with A-T.
The purpose of this study is to find out how safe and effective the study drug may be in improving the neurological symptoms that occur in patients with A-T.
The study consists of 2 parts, both lasting 6 months. In the first part of the study patients will be randomly assigned to receive either the study drug or placebo – that is a version of the treatment that contains no active drug.
Patients will be divided into three groups to receive either a lower dose-range (5-10 mg) or a higher dose-range (14-22 mg) or the placebo. Each patient will therefore have a 2 in 3 chance of receiving study drug and a 1 in 3 chance of receiving placebo. Neither the participant nor the doctor will know which study treatment has been assigned to them. This type of trial is called “double-blind” and is the gold standard for studying new drugs. Placebo-controlled trials are necessary to establish the benefits and risks of treatments and allow the EMA, FDA, or other regulatory agencies, to make decisions about whether or not a drug should be approved for marketing.
Following 6 months in the study, all patients who complete the designated procedures and tests will be eligible to continue in an additional 6-month study to collect information on the long-term safety and benefits of the study drug. Patients originally assigned to the study drug will continue on the same treatment, while patients on placebo will be progressively shifted to the study drug.
Once a patient completes the one-year study, they will have the option to continue receiving the study drug as part of a separate, on-going study. At this stage all patients will be receiving the drug.
Patients may be eligible to participate if they have been diagnosed with A-T, are at least 6 years old, weigh over 15 kg (33 pounds) and able to walk independently or walk with periodic use of a support. For more details about this study, including a list of requirements for entering the study, visit http://www.clinicaltrials.gov This study has the clinical trials identifier: NCT02770807
A total of about 180 people are being recruited to participate in this study worldwide. To locate your nearest study center, please click here. The study team will meet with you to discuss the study, review your medical and A-T history and perform screening tests to determine if you are eligible to participate.
Choosing to participate in a clinical trial is an important personal decision. The frequently asked questions section provide detailed information about ATTeST and clinical trials in general. In addition, it is often helpful to talk to a physician, family members, or friends about deciding to join a trial. The next step is to contact the study team and ask questions.
Participants in clinical trials can gain access to new investigational drugs before they are widely available, and help others by contributing to medical research. Participation in clinical trials can play an important role in getting new drugs approved for use in treating other patients.
In most cases, patients agree to be enrolled in clinical trials because they can gain access to new, experimental drugs or devices – especially if there are no other treatment options or if all of other treatment options have failed. Some patients may benefit from participating in a clinical trial if the investigational drug turns out to be effective.
In general, each clinical trial has inclusion and exclusion requirements that are intended to make the study more likely to demonstrate the effect of the treatment (such as excluding patients who are taking certain other drugs or who have other complicating disease conditions), or to protect patients by minimizing risks to certain populations (such as pregnant women). Additionally, a clinical trial for a specific drug is typically conducted at a limited number of institutions. Patients who live too far away from a research center may not be able to participate.
Drugs that are being tested in clinical trials may not end up proving to be effective, or they may have unknown, sometimes severe, side effects.
If you are considering participating in a clinical trial, you should carefully read all the information about the drug trial you are considering and ask questions about exactly what will happen during the trial, during a process known as “informed consent.” You are entitled to receive detailed information regarding what is known about the drug before you decide to enter a clinical trial, and you must provide a signed informed consent document indicating that you understand what will happen during the clinical trial before participating. You should also get advice from your healthcare professional.
General questions on clinical trials
Investigational or experimental drugs are either new drugs that have not yet been approved by the European Medicinal Agency (EMA), the US Food and Drug Administration (FDA) or other regulatory agencies or they are approved drugs that have not yet been approved for a new use, and are in the process of being tested for safety and effectiveness.
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it and the care expected while in a trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
A protocol is the study plan on which each clinical trial is based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial, the schedule of tests, procedures, medications, drug dosages and the length of the study. While in a clinical trial, participants following a protocol, are seen regularly by the research team to monitor their health and to determine the safety and effectiveness of their treatment.
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:
All clinical trials have guidelines about who can participate. The use of inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. 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.
Before joining a clinical trial, a participant must be eligible 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.
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental drugs are often compared with placebos to assess the experimental drug’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental drug.
The clinical trial team includes doctors, nurses and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.
Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the study staff.
There are risks, discomforts, and inconveniences associated with any clinical trial. You may have side effects from taking the investigational drug. Side effects are any undesired actions or effects of the investigational drug or treatment. Potential side effects will be discussed with you during the informed consent process; however, all of the risks associated with taking the investigational drug may not currently be known. Throughout the clinical trial you will be checked by the study team for side effects and it is very important that you tell the study doctor or nurse of any side effects that you have. Investigational drugs must be evaluated for both immediate and long-term side effects.
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.
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, while the control group is given either a standard treatment for the illness or a placebo.
Specific questions for the trial
This study is for research purposes only. It is possible that the symptoms your child experiences from A-T may improve because he or she is taking part in this study, but there is no guarantee that they will benefit in any way.
By participating in this study, you or your child may help gather information on the effectiveness, safety, and tolerability of the study drug, which may also help other patients in the future. After 12 months, you or your child may be offered an opportunity to participate in an additional extension study if they meet the criteria. During this extension study, they will continue to receive the active study drug.
You will be informed in a timely manner if information becomes available that might influence your willingness to continue participating in this study.
Most patients stopped the treatment at the end of the 6-month Phase II IEDAT trial since there were not planned extension of it. However, some went on receiving the drug under a compassionate treatment protocol and are still being treated with EDS today. As described in a recent publication by Leuzzi et al. 2015 (in Neurology, Neuroimmunology and Neuroinflammation), 4 patients continued to be treated with monthly EDS infusions for an additional 24 months. Their clinical outcome was compared with that of 7 age-matched patients who stopped the treatment after the first 6 infusions (IEDAT study). Patients in the extended study experienced a continuous neurological improvement with respect to their pretreatment status, whereas controls showed a progressive neurological deterioration (in accordance with the natural course of the disease) after the discontinuation of the treatment. The delivery system used proved to be safe and well-tolerated, and none of the side effects usually associated with the chronic administration of corticosteroids were observed during the entire trial. As suggested by the authors, these promising preliminary results call for a large-scale, controlled study looking at the extended treatment of patients with A-T with dexamethasone-loaded red blood cells.
The drug should improve the neurological symptoms of the disease, general quality of life and adaptive behavior.
In a previous phase II trial (IEDAT) in 22 patients with A-T, EDS treatment for 6 months led to a statistically significant improvement in the primary measure of effectiveness, the ICARS scale, which assesses key neurological symptoms of the disease. Other statistically significant benefits of the EDS treatment were also seen in secondary measures, such as kinetic function (movement), quality of life, adaptive behavior and eye movements.
The study drug was generally well tolerated in this subject population, with only two patients discontinuing prematurely due to adverse events. More details can be found in the paper by Chessa et al. 2014 in the Orphanet Journal of Rare Diseases.
The EDS procedure ensures that after a rapid, short-lived peak, the drug is released at low and slowly-declining concentrations over the course of approximately 4 weeks. This is different from the daily administration of systemic steroids, which has to be done at higher doses. Dexamethasone is also more effective in penetrating the central nervous system than other steroids such as prednisolone. These factors suggest that this method of administration may be associated with beneficial effects without the risk of steroid side effects.
The ATTeST trial will last a total of 12 months for each participant. Following completion of 6 months in the study, all patients who complete the designated procedures and tests will be eligible to continue in an additional 6-month study, to collect information on the long-term safety and benefits of the study drug. Patients who were originally assigned to receive the study drug will continue on same treatment, while patients on placebo will be progressively shifted to the study drug. Once each patient has completed one year in the study, and is eligible, he/she will have the option to continue receiving the study drug as part of a separate study (with no placebo control group).
“Double-blinded” means that none of the patients, the parents or study teams will know if the patient is assigned to the drug or placebo group.
In order to determine whether the study drug improves the neurological symptoms of A-T it is necessary to compare the results in participants receiving EDS with those in participants who are receiving a placebo. However, following completion of 6 months in the study, all patients who complete the designated procedures and tests will be eligible to continue in an additional 6-month study to collect information on the long-term safety and benefits of the study drug. Patients originally randomized to receive the study drug will continue on the same treatment, while patients on placebo will be progressively shifted to the study drug. At the completion of 12 months in the study, all patients will be receiving the study drug. Each patient who has completed one year in the study will have the option to continue receiving the study drug as part of a separate, on-going study.
More complete inclusion and exclusion criteria are available at www.clinicaltrials.gov and will be explained in detail by the doctors conducting the study, but here are some key considerations. Potential participants must:
Consent forms are legal documents that can only be signed by adults, and assent forms give minors (i.e., children) the opportunity to convey their own independent decision to participate in research.
The decision as to whether a patient is eligible for the study is ultimately made by the principal investigator and the study team at the study site. The team can also help each patient and family consider the possible risks and benefits to them.
Participants must be at least 6 years of age. Providing that the person meets all other criteria, there is no upper age limit.
The patient may be in this research study for up to 13 months (including the initial screening visit). The patient will be asked to visit the study site at least 16 times over this 13-month period. If needed, the study doctor may ask patients to come to the study site for additional visits.
Placebo-controlled trials are necessary to establish the benefits and risks of treatments. Patients who are randomized to receive the placebo will receive the EDS procedure without the investigational drug, dexamethasone.
During the 30-day screening period (which is considered a washout period from previous treatments), any previous treatments with other corticosteroid drugs will be withdrawn. Other restrictions may also apply. More details will be provided by the study team at the site.
Unfortunately, we will not be able to include every patient immediately in this trial, and for that we are truly sorry. However, we are committed to completing the Phase 3 trial as quickly as possible so that if EDS proves to be safe and effective, it can be made available to the A-T community in the shortest period of time. We know that each decision made about our clinical program impacts patients and families around the world. However our overriding aim is to learn definitively and as quickly as possible whether or not EDS is a safe and effective treatment option for people with A-T.
Yes he/she can participate in any of the selected study sites unless local legal restrictions apply.
The study drug, administered through the EDS, is an approved drug and is available by prescription for treating a number of chronic (long-term) inflammatory diseases. Patients with these diseases have to take large doses of the drug orally or by injection for long periods of time and this can result in serious side effects.
A list of most common side effects of the study drug will be found in the study informed consent form.
In the course of clinical studies conducted in Europe and the United States, the EDS has been used to give the study drug to 209 human subjects. Over 1800 infusions prepared using the EDS have been given to those participating in these studies, with some having received the treatment over two years. No toxicity related to the administration of the study drug using the EDS has been reported.
However, because the EDS is a new method of delivering the study drug, all its side effects may not be known. You must tell the study staff about any side effects that your child develops.
Every clinical trial must be approved and monitored by an Institutional Review Board (IRB) or Ethics Committee (EC) to make sure the risks are as low as possible and are worth any potential benefits. An IRB or EC is an independent committee of physicians, statisticians, community advocates and others that ensures that a clinical trial is ethical and that the rights of study participants are protected.
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is regulated by the government 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 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.
For ATTeST, EryDel has added an additional independent Data Safety Monitoring Board. This Board is composed of four independent experts who will periodically monitor the safety results and will be able to stop the trial if any safety concerns are detected.
Any research has some risks, which may include things that could make your child feel bad or uncomfortable. In a research study like this one, not every risk or side effect may be known or can be predicted. Each person’s reaction to a test, drug, or procedure may be different. Your child may have a side effect or be at risk for symptoms, illnesses and/or complications that could not be predicted by the study doctor or the makers of the EDS.
In addition, some possible risks may be associated with drawing blood from your child arm (such as pain, swelling, bruising and, on rare occasions, infection at the injection site) or with blood infusion (causing fever, chills, nausea, hives, rash, itching, swelling of the face or neck, difficulty breathing and low blood pressure). Furthermore, there may be some discomfort with the venipunctures, and a bruise may appear at that site for a few days.
We only have limited data regarding the use of the EDS in the treatment of A-T so we are unable to draw any conclusions on this. To avoid any potential negative effect for patients in coming off the medication on completion of the ATTeST trial, patients may be offered the opportunity to participate in an additional extension study in which they will continue to receive the active study drug.
On average there is one needle stick per visit. This is required both for loading the study drug or placebo with the EDS procedure and to provide samples for the laboratory tests required to analyse all safety parameters of the study.
EryDel has selected study sites with well-prepared staff, who are experienced in working with people who have A-T.
The EryDex System (EDS) is used to deliver dexamethasone to the patient by loading dexamethasone sodium phosphate (DSP), an already marketed drug, into the patient’s red blood cells, which are then reinfused into the patients.
In order to provide the patient with the study drug or placebo, about 50 mL of his or her blood will be collected. In brief, the Erydex system will add the drug to the red blood cells derived from the sample. The red blood cells are then infused back into the patient, where the drug is slowly released from the cells into the bloodstream. As small amounts of study drug or placebo are released gradually into the patient’s body over a long period of time (approximately one month), this procedure is expected to cause fewer side effects than when the drug is given as a tablet or an injection.
The study will be conducted at centers in North and Central America, Europe, North Africa, Asia and Australia. A list of study sites can be found here.
Possibly. Your ability to attend and participate in one of the study sites is dependent on local laws and regulations. Each situation would need to be addressed on a case-by-case basis by the clinical trial Principal Investigator. We encourage you to contact a study site to discuss your specific circumstances.
Start dates are not fixed yet and will depend on specific factors at each study site. EryDel has initiated all the activities required to receive the approval from each local Institutional Review Board. You can find the latest information by contacting the nearest study site directly.
Because the study complies with all applicable international, federal, state and local laws and regulations regarding the conduct of its clinical trials, including good clinical practice, international travel restrictions may apply. In addition, to ensure clinical study integrity, there may be relevant ethical and practical issues to consider when enrolling A-T subjects. The eligibility of patients will be determined by the study clinical trial team at each study site, who may consider factors such as travel distance, medical insurance and their specific institutional policies regarding international patients.
Yes, EryDel is planning that patients who complete the 12-month trial will be eligible to participate in an open-label trial extension (where everyone receives the drug).
This will be established by Regulatory Authorities on the basis of the results of this trial. If the investigational drug of the ATTeST trial proves to be effective and safe and receives regulatory approval and market authorization in your country, your doctor will be able to prescribe the treatment. It is the regulatory authority who will decide whether any restrictions on usage of the drug are needed.
If the drug proves to be effective and safe, and it receives regulatory approval and market authorization in your country, doctors will be able to prescribe it to treat A-T.
In principle not, but it would need to be assessed on a case by case.
Unfortunately we currently have limited evidence that the study drug will have a benefit in patients that cannot any longer walk. This is the reason why only patients with autonomous gait or at least helped by periodic use of a support are eligible to participate in the ATTeST trial. If the drug is found to be safe and effective on participants in the trial, it is possible that in future it could be shown to have a positive effect in those who can no longer walk.
We have currently no other trials ongoing with EDS.
Yes, we have produced a patient information sheet and informed consent form using language that children should understand. We have also prepared a specific section (4Kids) in this web site.
There will be a patient reimbursement program based on travel to the nearest available study site. The details and applications may differ from country to country. Detailed information will be provided by the study site.
If you are interested locating the study site nearest to you please use the map below.
By clicking to the pins, contact details of each Study Center will show up
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