CLN2 Cerliponase Alfa (Brineura) Treatment Survey

There is one qualification to participate in this study:
Your child has Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease (choose "yes" or "no") to answer this question.

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Yes

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Information Sheet

You are invited to participate in a research study because your child has been diagnosed with CLN2 Batten disease. The goal of this research study is to learn more about families' experiences with Cerliponase Alfa (Brineura) and decision-making around use of this treatment. Your child does not have to be receiving Brineura in order for you to take part in the survey. We are interested in hearing from all affected families, whether or not your child has ever received this treatment.

This study is being conducted by researchers at the University of Rochester Batten Center:

Principal Investigator (PI): Michael Lyashenko, First Year Medical Student, University of Rochester School of Medicine and Dentistry, URMC.

Co-PI, supervising faculty: Heather Adams, PhD, Department of Neurology, Division of Child Neurology, URMC.

If you choose to participate in this study, you will complete an online survey about your decisions surrounding the use of Cerliponase Alfa (Brineura) as a therapy for your child's CLN2 disease, and what factors may have influenced those decisions.

This study will look at the experiences of children with CLN2 disease and their parents/caretakers with respect to the use of and decision-making about Brineura. To better understand these aspects of the treatment, we have created a survey that will assess your and your child's experiences. Questions in the survey will ask about topics such as whether your child is on Brineura, has ever been on Brineura, their experiences while on Brineura, your/your family's experiences while your child is/was on Brineura, reasons for choosing to use or not use Brineura as a treatment, and difficulties experienced while obtaining Brineura. We will also ask you to provide some information about your affected child (for example, their symptoms and if/how they have changed over time) and about your child/family demographics. This survey will be completed online. It will take approximately 30 minutes. You may find some of the questions upsetting or uncomfortable. You may skip any questions you don't want to answer.

You and your child will not benefit from being in this research study.

We estimate that approximately 50 participants will take part in this study. The survey will be available for 6 months.

Although absolute confidentiality cannot be guaranteed, all reasonable efforts will be made to prevent loss of confidentiality. Your answers will be anonymous - we will not collect any information that could specifically identify you or your child. Your survey answers will be coded with a confidential identification code. Your survey responses will be collected, recorded, and stored using REDCap (Research Electronic Data Capture), which is on a secure network at the University of Rochester. Only members of the study team will have access to your survey responses. General results of this research may be presented at conferences or in publications, but there will be no information presented that could identify you or your child.

Your participation in this study is completely voluntary. You are free not to participate or to withdraw at any time, for whatever reason. Any information already collected in the survey will continue to be used for the research. However, if you do not wish for your information to be used after withdrawing, they may request this of the investigator/study team. No matter what decision you make, there will be no penalty or loss of benefits to which you are otherwise entitled.

For more information or questions about this research you may call Dr. Heather Adams, PhD: 585-275-9330 (Co-Principal Investigator).

Please contact the University of Rochester Research Subjects Review Board at 265 Crittenden Blvd., CU 420628, Rochester, NY 14642, Telephone (585) 276-0005 or (877) 449-4441 [insert country code (001) if applicable] for the following reasons:

You wish to talk to someone other than the research staff about your rights as a research subject;
To voice concerns about the research;
To provide input concerning the research process;
In the event the study staff could not be reached.

By continuing to the next section and completing the survey, you are consenting to participate in this study.

*Please print or save a copy of this form for your records*

You have selected an option that triggers this survey to end right now.

To save your responses and end the survey, click the 'End Survey' button below. If you have selected the wrong option by accident and/or wish to return to the survey, click the 'Return and Edit Response' button.