Living with CIDP? You Are Not Alone

Through clinical research studies, also called clinical trials, scientists and doctors can determine whether a new medical strategy, drug, or device is safe and effective for people. New treatments cannot be authorized for clinical use without clinical research studies, which enroll potential study participants like you to evaluate the safety and effectiveness of these treatments. Taking part in a research study is voluntary and is not part of your regular health care. 

When an investigational medication is being tested in a clinical research study (a study medication), it is tested in stages, or “phases.” Phase 1 studies include a small group of people, often without any medical conditions, to gather information on the safety, dosing, and any side effects of an investigational medication. Phase 2 studies measure the safety and efficacy of an investigational medication in a group of people living with a certain disease or condition. Phase 3 studies measure the safety and efficacy of an investigational medication in a larger group of people for a longer period of time and compared to commonly used medications (standard of care) or placebo. A Phase 2/3 study combines aspects of Phase 2 and Phase 3 studies into a single study. Phase 2/3 studies are often used to study a rare disease like CIDP.

Each phase is designed to learn more about whether and how the study medication works and how safe it is.  The Phase 2/3 study is submitted to regulatory agencies (e.g. FDA) for review and approval before the investigational medication can be approved and authorized to be prescribed to patients.

A placebo is a treatment that looks exactly like the investigational medication but contains no active drug. In clinical research studies, researchers often compare an investigational treatment to a placebo (no active drug). All of the participants receive the same level of care, regardless of whether they are assigned to receive the investigational medication or the placebo. This helps researchers better evaluate if the investigational medication is safe and effective, when compared to placebo.

Participants will be closely monitored during the entire study, including site visits after receiving the investigational medication. Participants will be asked about symptoms throughout the study. The study site doctor and/or staff will also check in with participants over the phone between study site visits. If symptoms worsen, a study doctor will evaluate and may treat the participant with standard CIDP treatments. Participants may contact the study site at any time if there are questions or concerns.

Clinical research helps doctors and scientists determine if an investigational medication or treatment is safe and/or effective for use in affected patients to potentially treat a condition, disease or disorder. Clinical studies often require hundreds or thousands of volunteers to participate in a single study, sometimes thousands are needed to obtain reliable information. 

You can check if you are potentially eligible for this study by completing a set of pre-screening questions. You can find these on this website by clicking the “See if You Pre-Qualify” button.  A study doctor will contact those who express interest to further review eligibility for the study and to provide more information on what participation will entail.

If you participate in this study, the study doctor and other study site staff will collect your personal information. This will be reviewed with you by the study team, and you will be able to ask any questions you have before you sign the Informed Consent document and agree to participate. Personal information that is collected after signing the Informed Consent document may include: - Your name, address, and contact details - Your age, sex, and ethnic and racial background - Your lifestyle information - Your medical history details - Your study treatment and your response to these treatments - Data resulting from testing your biological samples If you complete the online questionnaire to see if you may pre-qualify for the study, you will also be asked to give permission for your information and contact details to be shared with a nearby study site. No information will be shared without your permission. 

You can agree to be in the study now and change your mind later at any time and for any reason. You can withdraw from the study either verbally or in writing, at any time, and your decision will not change your regular care from your doctors.

You can talk to the study doctor or study staff first before making this decision.

Not all individuals who complete the pre-screener will be eligible to participate in the study.  You will be notified if you are not eligible for the study, based on your responses. You can speak with your doctor about other options or clinical research studies that you may be eligible to participate in. Please view the Resources section to see other CIDP resources you may consider.