MDA Kickstart Patient Engagement

Protocol No.: None
WCG IRB Protocol #20251842

Sponsor: Muscular Dystrophy Association (MDA)

Investigator: Angela Lek, PhD

1016 W Jackson Blvd Ste 1073

Chicago, Illinois 60607

United States

STUDY RELATED-

PHONE NUMBER(S): 800-572-1717

Thank you for your interest in learning more about how to be involved with MDA’s Kickstart Program.

As part of MDA’s Kickstart Program, we are seeking to better understand the disease progression and burden of Congenital Myasthenic Syndrome (CMS) caused by mutations in the choline acetyltransferase (CHAT) gene. CHAT-CMS is the pilot condition selected for MDA’s Kickstart Program. It is a disorder that causes muscle weakness and is often associated with episodes of respiratory arrest, with no effective treatments to date.

If you are interested in participating in patient engagement initiatives as part of our Kickstart Program, please answer the pre-screening questions below to determine your eligibility to participate in our future studies. If eligible, you will be asked to complete a consent form to allow MDA to contact you.

Please answer the following eligibility questions:

1. Select the option that applies to you:

I am a person living with CHAT-CMSI am a primary caregiver of a person living with CHAT-CMSNone of the above options apply

2. Are you 18 years of age or older?

You are not eligible to participate in future study opportunities. We are looking for the following requirements for study participation:

1. You must have a diagnosis of CHAT-CMS or be the primary caregiver of a CHAT-CMS patient

2. You must be 18 years of age or older

If you know someone who may be eligible, please refer them to this website.

Next, we’ll ask you to read through the informed consent form. This document provides information to help you decide whether or not you would like to be contacted by MDA as part of the Kickstart Program.

As part of MDA’s Kickstart Program, we are looking to learn more about the patient and caregiver experience as it relates to CHAT-CMS.

Your participation is voluntary and there will be no costs to you. You will not be paid for giving an eConsent to be approached. There are no guaranteed benefits to you or your household because of participation in this initiative.

If you do not want to be approached or decide to end your participation, there will be no penalty to you.

Every effort will be made to safeguard the information you provide but there is a chance that it could be inadvertently disclosed or inappropriately accessed. We protect your information from disclosure to others to the extent required by law. We cannot promise complete secrecy.

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.

You are invited to give consent for a qualified member of MDA’s Kickstart team to contact you about participating in patient engagement initiatives.

Are you willing to learn more about MDA's Kickstart program for CHAT-CMS? (Select One)

YesNo

If yes, you will be contacted by MDA to be provided information about our Kickstart Program patient engagement initiatives. Please include your contact information below.

First Name of Patient with CHAT-CMS

Last Name of Patient with CHAT-CMS

Patients Date of Birth

Caregiver Information is required if Patient is under 18

First Name of Caregiver

Last Name of Caregiver

Relationship to Patient

Country

State of Residence

Contact Phone Number

Alternate Phone:

Contact Email

Preferred Method of Contact

What is the best time to contact you (check all that apply)?

MorningAfternoonEvening

How did you find out about MDA's Kickstart Program?

Other

Name of Diagnosing Physician (if applicable)

Diagnosing Physician's Institution (if applicable)

By completing this eConsent to be approached, you will be automatically registered for future contacts by a qualified MDA Kickstart team member.

You have been made aware of the reasons why the contact information is needed and the risks and benefits of consenting or refusing to consent.

I accept

For questions about MDA’s Kickstart Program, you may contact the following email: resourcecenter@mdausa.org or talk to the research team at the phone number listed in this document.

Over 18

Over18

age