FAQ
Yes, previous studies in A-T suggest that agents such as steroids may help patients with Ataxia-Telangiectasia.1,2 A-T symptoms are assessed using specific neurological scales that measure the effects of A-T on patients’ posture, gait, movements (including the eyes) and speech. The corticosteroid used is already approved for the treatment of various long-term inflammatory conditions but not specifically for Ataxia-Telangiectasia.
Before each treatment, around 50 ml of blood is taken from you or your child. The subsequent infusion consists of around 70-80 ml of liquid, which contains your or your child’s red blood cells – loaded with the active substance or the placebo. It is administered via the arm vein or, if available, via a central venous catheter (a thin tube in a large vein). This procedure takes about 40 minutes.
The study participant will receive this new potential treatment at the study site approximately every 28 days. During the treatment period, EryDex will be given in a total of 6 infusions to you or your participating child.
Yes. All participants are assigned to either a control or experimental group. There is, therefore, a 50% chance that you or your child will receive the placebo, not the active ingredient.
Upon study completion and if you or your child are willing to continue treatment and the study doctor considers you or your child eligible to continue treatment there will be an opportunity to continue in a study where everyone receives the active substance.
Side effects can always occur when taking medication. The frequency of side effects of approved medicines is primarily known and is stated in the package leaflet. In the case of investigational drugs that are still undergoing clinical trials, adverse events can occur that were not foreseeable. For this reason, study participants are monitored closely in order to identify any potential issues and act quickly as needed.
This study has been reviewed and authorized by the responsible federal authorities and ethics committees. In addition, the study doctor will discuss the possible known side effects with you in detail and what to do in the event of adverse events.
The A-T Study aims to find out whether the potential new treatment works for treating the neurological symptoms of A-T and will be assessed based on patients’ neurological symptoms or using specific neurological examinations and scales: The effects of A-T on patients’ posture, gait, movement (including the eyes), and speech will be measured. Study participation is voluntary, and you or your child can freely withdraw at any time.
Upon study completion and if you or your child are willing to continue treatment and the study doctor considers you or your child eligible to continue treatment there will be an opportunity to continue in a another study where everyone receives the active substance.
The study is currently in phase III, and the potential new treatment is being investigated in at least 106 people with a genetic diagnosis of A-T. The A-T Study is a randomized, placebo-controlled, multicenter, and double-blind study.
Placebo-controlled means that the effect of the investigational drug is tested in comparison to a placebo. A placebo looks exactly like the drug being tested but contains no active ingredient.
Randomized means that, just like flipping a coin, chance decides whether the patient receives the investigational drug or the placebo. This way, researchers avoid any influence on allocation. Randomization takes place at the beginning of the treatment phase.
Double-blind means that neither the members of the study team nor the participants or their relatives know which treatment they will be assigned to. This means that no one can tell you whether you or your child is receiving a placebo or an active substance. The purpose of “blinding” in studies is to prevent members of the study team or the study participants from influencing the results. For example, the mere expectation that the medication should help can affect a test person’s statements about their symptoms.
Multicenter means that the study is conducted at several study centers, which has the advantage that participants can be recruited from different geographical areas.
Per government regulations, most new drugs or therapy must first be tested in clinical trials before it can be released for general treatment. The studies serve to test the efficacy and safety of the new drug. Strict guidelines apply, and study physicians closely monitor the participants. For patients, participation in clinical trials means that they have the opportunity to receive a new active ingredient to treat their disease in a clinical trial setting.
Clinical trials can be categorized into four phases:
Phase I: An investigational drug is administered for the first time to a small group, often healthy volunteers. This starts with a very low dose, which is then carefully increased. The primary aim is to investigate safety.
Phase II: A somewhat larger group of patients receives the investigational drug. In addition to safety and tolerability, preliminary efficacy is also assessed.
Phase III: The investigational drug is tested in larger, often international, clinical trials that may involve several hundred or thousands of patients. Safety, tolerability, and efficacy are all assessed. With positive results from these studies, companies can apply for marketing authorization for the investigational drug. If successful, it becomes an officially approved drug that is then available to patients.
Phase IV: Sometimes, further studies may be conducted after the drug has been approved to obtain more information, e.g., on side effects, clinical benefits, and safe use in everyday clinical practice.
With our questionnaire, you can easily check commitment-free whether you or your child may be suitable for the study and can participate.
Participation in the A-T Study lasts approximately 8 months. Participation includes at least 8 visits to the study site in person: 1 visit for the preliminary examination, 6 visits for the administration of the infusions, and 1 visit for the safety follow-up.
In addition, there will be 3 appointments during the treatment phase, 2 of which will be by telephone and 1 on-site or by phone.
No, you do not have to pay anything to take part in the study. The study sponsor bears the costs of the medication administered in the study and those of all examinations and laboratory analyses. A specialist travel company arranges all travel and accommodation. You will not have to pay for any travel or accommodation directly for you and or your family, this is paid for by the Sponsor directly to the travel company.
Your health insurance or that of your child does not influence participation in the A-T Study. Costs related to examinations and treatments of the study will not be billed to your health insurance, as the sponsor covers all clinical trial costs. In addition, Quince Therapeutics is obligated to arrange participant insurance. It covers possible risks of study participation.
The new potential treatment was developed by researchers at Quince Therapeutics, based in San Francisco, USA. The biotechnology company specializes in developing innovative therapeutics for rare diseases such as Ataxia-Telangiectasia.
Quince Therapeutics has commissioned Trials24 GmbH to assist with the recruitment of suitable participants for the A-T Study. Trials24 GmbH is based in Munich, Germany. It is a specialized service provider and supports pharmaceutical companies in finding participants for clinical trials to make innovative medicines available to patients more quickly.
You can simply check commitment-free through the questionnaire whether you or your child may be suitable for participation in the study, simply and without obligation.
If you have any further questions, you can contact our Patient Information Service. You can reach them by email at info@a-t-study.com, via the contact form, or Monday to Friday from 9 a.m. to 6 p.m. by telephone +1 415 635 0310.
Use our commitment-free questionnaire to find out whether you or your child are eligible to participate in the study.
Do you have any questions? Then give us a call!
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