Help speed the pace of research towards treatments and a cure for FA by participating in a clinical trial or study today. Active studies are listed on the right.
To stay informed about opportunities to participate in clinical research studies, please sign up for the Global FA Patient Registry.
Overview of Gene Therapy for Friedreich's Ataxia
Gene Therapy Preparedness
Here are some questions that are frequently asked about participation in clinical trials:
A: A clinical study involves research using human volunteers to add to medical knowledge. There are two main types of clinical studies, clinical trials and observational studies.
A clinical trial evaluates a specific intervention according to the research plan or protocol created by the investigators. These interventions may be medical products such as drugs or devices, procedures, or changes to participants' behavior (for example, diet). Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants.
In an observational study, investigators assess health outcomes in groups of participants according to a protocol or research plan. Participants may receive interventions, which can include medical products or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator.
A: By participating in a clinical trial, you can advance medical research and help shorten the timeline to develop treatments and a cure for FA.
Participating in a clinical study contributes to medical knowledge. The results of these studies can make a difference in the care of future patients by providing information about the benefits and risks of therapeutic, preventative, or diagnostic products or interventions. Some trials may provide participants with the prospect of receiving direct medical benefits, while others do not. A potential participant should also discuss these issues with members of the research team and with his or her usual health care provider.
A: Eligibility criteria vary from study to study, so it's best to contact the study coordinators directly to see if you are eligible for a specific trial.
A: Here is a list of questions you should ask study investigators when deciding to participate in a trial:
- What is being studied?
- Why do researchers believe the intervention being tested might be effective? Why might it not be effective? Has it been tested before?
- What are the possible interventions that I might receive during the trial?
- How will it be determined which interventions I receive (for example, by chance)?
- Who will know which intervention I receive during the trial? Will I know? Will members of the research team know?
- How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
- What will I have to do?
- What tests and procedures are involved?
- How often will I have to visit the hospital or clinic?
- Will hospitalization be required?
- How long will the study last?
- Who will pay for my participation?
- Will I be reimbursed for other expenses?
- What type of long-term follow-up care is part of this trial?
- If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
- Will results of the study be provided to me?
- Who will oversee my medical care while I am in the trial?
- What are my options if I am injured during the study?
- Will I have to stop taking any of my current medications or supplements to participate in this trial?
- Will I be allowed to participate in another clinical trial if another one comes up during this trial? Do you recommend another clinical trial for me over this one?
A: Follow the rules!
Compliance with study visits, dosing of the intervention, diaries, etc…
Communication with the study investigators, especially about any possible side effects. Report any time you visit a hospital, ER, outpatient clinic. You should not be determining what is side effect or not, since that is the responsibility of the study investigator.
Refrain from conducting your own experiments as this will invalidate any possible result that the study was designed to evaluate.
Maintaining Confidentiality. Communicating your impressions of study experiences can bias study investigators and participants, compromising the integrity of the study.
Things you can talk about: Traveling to the study site, the rules of the study, the study coordinators and investigators, other participants in the study.
Things you cannot talk about: Whether you think you have placebo or drug, whether you feel the drug is working, how you feel on the drug.
A: You can withdraw or decide not to participate at any time – no questions asked! If you withdraw during the active phase, it is very important to participate with the withdrawal visit to properly close your participation.
Adapted from http://www.clinicaltrials.gov/ct2/info/understand.
Find up-to-date clinical trial information for Friedreich's ataxia at clinicaltrials.gov.