Informed Consent for Participating in the Batten Disease CLN1 Registry

TITLE: Batten Disease CLN1 Registry
PROTOCOL NO.: None
WCG IRB Protocol #20241428
SPONSOR: Collaborations Pharmaceuticals, Inc.
INVESTIGATOR: Sean Ekins, PhD, DSc
840 Main Campus Drive
Lab 3510
Raleigh, North Carolina 27606
United States
STUDY-RELATED PHONE NUMBER(S): 215-687-1320 (24 hours)
Your participation in this study is voluntary. You may decide not to participate or you may leave the study at any time. Your decision will not result in any penalty or loss of benefits to which you are otherwise entitled.

If you have questions, concerns, or complaints, or think this research has hurt you, talk to the research team at the phone number(s) listed in this document.

Definitions

For the purpose of this Consent form, “the patient” refers to the person with CLN1 Batten disease. “Primary caregiver/parent/guardian” refers to the person who legally represents the CLN1 Batten disease patient. Batten Disease CLN1 registry information will be collected on patients with CLN1 Batten disease only. “You” refers to the person providing the information, who may be the primary caregiver, parent, or a family member or guardian who is legally responsible for the care and health of the patient.

Purpose

This Batten Disease CLN1 registry has been created to enable research on this rare disease which affects approximately 100 people globally. The registry collects and stores basic patient medical information for use in medical research. The purpose of Batten Disease CLN1 registry is designed to:

  1. Identify families with children currently living with CLN1.
  2. Obtain basic contact information on the primary caregiver and disease history.
  3. Assist in recruitment for future natural history and clinical studies.
  4. Analysis of the deidentified questions for future publication.
The primary caregiver/patient/guardian provided information email and contact details will be stored separately in this registry and will be used to contact them for future CLN1 Batten disease natural history and clinical trials which will develop new treatments. Collaborations Pharmaceuticals, Inc are studying CLN1 Batten disease and need accurate information on where these patients are located and to understand how the disease affects them. There is no cost to you to participate. We plan to collect information from participants for at least one year before reporting our findings. The registry may continue longer depending on support of this project which is currently funded by Collaborations Pharmaceuticals, Inc.

What will happen if you join this registry?

If you agree to join this Batten Disease CLN1 registry, you will be asked to provide basic medical information on your (or more likely the patient's) disease and diagnosis which should take about 10 minutes. The Batten Disease CLN1 registry aims to share collected deidentified medical information with the principal investigator at Collaborations Pharmaceuticals, Inc. while protecting your privacy. One way the Batten Disease CLN1 registry protects your privacy is to remove your name, address and other “identifying” information from your patient related information before providing it to the principal investigator. This information is “de-identified” because it has had all personal identifiers removed including your name, address, or other information that identifies you or your family. The information you provide to the registry will be stored on secured computers and servers and protected with encryption and passwords.
The Batten Disease CLN1 registry will not share your identifiable information with anyone (unless you give your permission to share it). Only approved researchers will be allowed to see the de-identified information. The information collected and compiled by the Batten Disease CLN1 registry belongs to Collaborations Pharmaceuticals, Inc. who maintains the information contained within the registry.

Your participation is voluntary

Providing information to the Batten Disease CLN1 registry is voluntary. You do not have to contribute information. If you do participate, you can withdraw from the Batten Disease CLN1 registry mailing list at any time and for any reason. To ensure absolute accuracy, primary caregiver/guardians/parents/patients who decline to give their consent will not be included in the Batten Disease CLN1 registry.

Right of withdrawal

Should a primary caregiver/guardian/parent/patient change their mind and wish to withdraw from the Batten Disease CLN1 registry in which they are registered, they will be free to do so without having to provide any explanation. Simply contact the approved researcher (sean@collaborationspharma.com) and your name and contact details will be removed from the database. Deidentified health information that has already been shared cannot be retrieved or removed.

Anticipated benefit

Participation in the Batten Disease CLN1 registry is not likely to benefit you (or the patient) personally, medically or financially. However, participation may help members of your family and others with CLN1 Batten disease and/or patients with CLN1 Batten disease by increasing the understanding of your condition. Having an available registry of information about CLN1 Batten disease may help speed up research. Such research could eventually help researchers to learn whether or how treatments work, or help medical professionals improve how they treat CLN1 Batten disease. Participants may receive information about opportunities to participate in research and clinical trials, as well as information about medical advances and other news from the registry.

Risks of participating

There is minimal risk in taking part in the Batten Disease CLN1 registry. The registry may ask you to answer questions that can be sensitive and you may feel uncomfortable answering. You do not have to share any information that you do not want to share. Another possible but unlikely risk is potential breaches in the computer system. In the event the there is a breach in the Batten Disease CLN1 registry computer system, you will be notified.

Alternatives to participating

Your alternative is to not take part in this research.

Participation of under-aged and adults unable to consent

Batten Disease CLN1 registry information will be collected on patients (who are diagnosed with CLN1 Batten disease) via their primary caregiver/parent/guardian. Patients over the age 18 who understand the consent form and legally provide their own consent (and thus do not have a legal guardian) are eligible to join the registry on their own. Otherwise, the legal guardian or parent of the patient must sign the consent form for the patient to join. When a minor participant becomes 18 (and if they are able), consent will be obtained directly from them for continued participation.

Other common questions

Is there payment for participation?

You will not be paid for being in this study.

What happens to the information collected for this research?

Your private information and your medical record will be shared with individuals and organizations that conduct or watch over this research, including: • The research sponsor • People who work with the research sponsor • The Institutional Review Board (IRB) that reviewed this research We may publish the results of this research. However, we will keep your name and other identifying information confidential. We protect your information from disclosure to others to the extent required by law. We cannot promise complete secrecy.

Who do I contact with questions?

If you have any questions about the registration process or about participation in the Batten Disease CLN1 registry, please contact the Approved researcher at 215-687-1320 (24 hours) or sean@collaborationspharma.com. To report concerns about your participation in the registry, you may contact the registry at sean@collaborationspharma.com. To inquire about your rights as a participant in the registry, you may also contact sean@collaborationspharma.com.

This research is being overseen by WCG IRB. An IRB is a group of people who perform independent review of research studies. You may talk to them at 855-818-2289 or clientcare@wcgclinical.com if: • You have questions, concerns, or complaints that are not being answered by the research team. • You are not getting answers from the research team. • You cannot reach the research team. • You want to talk to someone else about the research. • You have questions about your rights as a research subject.