You (“you” refers to you or your child throughout this consent form) are invited to take part in a retrospective observational research study using historical medical data. You are invited to join this study because you (or your child) have been diagnosed with chronic forms of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease Type B, or A/B.  

This document describes the purpose of the study and how your (or your child’s) data will be used. Please read the following information carefully.

If you are the parent or legal guardian of a child (i.e., under 18 years old) who has been invited to take part in this study, please review the rest of this document as it would pertain to your child. You have the choice to consent your child to participate and to contribute your child’s medical records data to this study.

The investigator is being paid by the sponsor (the company paying for this study) to conduct this research study.

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This observational research study looks at existing data that has already been collected and kept in your medical records from routine medical care. No new medical data about you will be collected in this study. Medical records (including but not limited to labs, medications, and doctor’s notes) from all participating patients going back as far as medical records are available will be gathered and extracted for analysis.

The purpose of this retrospective observational research study is to use medical records data to better understand the health of people living with ASMD in the U.S. The medical records data may be used to supplement findings from other studies and clinical trials.

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Sanofi Genzyme is funding the study. PicnicHealth is conducting this study on behalf of Sanofi Genzyme.

Yes. This study has been reviewed by IntegReview IRB, an institutional review board that is responsible for protecting the rights, safety, and well-being of patients who take part in research studies.

Up to 30 male and female patients, 21 years of age and younger, will participate in this study.

It will take approximately 10-15 minutes to finish registering for this study. You will not need to participate further in any activity.  The study will continue for 1 year.

If you agree to participate in this study, you will be asked to:

• Authorize PicnicHealth to collect your medical records on your behalf by signing this informed consent form
• List your healthcare providers (doctors, hospitals, and/or clinics)

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• You will be asked to sign this Informed Consent Form and a medical record release form. Only then will data from your medical records be extracted for analysis as part of this study.
• Your historical medical records data (i.e., at least 12 months of medical records before the date you enroll in this study) will be contributed to the study. If you are a participant in the olipudase alfa clinical trials ASCEND (NCT02004691), ASCEND-Peds (NCT02292654), or Long-Term Safety: LTS13632 (NCT02004704), only the medical records data before the date you enrolled in the clinical trial(s) will be contributed to the study.
• You will have access to PicnicHealth’s record collection services for 1 year from the time that you enroll in the study.  As part of these services, PicnicHealth will:

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1. Collect your medical records on your behalf from the healthcare providers that you authorize
2. Digitize your medical records so that you can access them online through your PicnicHealth account
3. Enable you to electronically share your medical records with your doctors, caregivers, and family.

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• After 1 year from the date that you enroll in this study, you will still be able to access your existing medical records through your PicnicHealth account.
• Personally identifying information (PII), such as your name or date of birth, will be removed before any analyses are being performed.  The data without your personally identifying information are referred to as “de-identified data.”  
• Your de-identified data will be aggregated (combined) with those of the other study participants and shared with Sanofi Genzyme who will analyze it to better understand the health of people who are diagnosed with ASMD.  
• You can request that your data be deleted from PicnicHealth at any time, but your data cannot be deleted from the study once it has been collected.

The following information will be extracted from your medical records:

• Demographics (gender, date of birth, etc.)
• Medical history (other diseases you may have or have had)
• Results of physical examinations
• Vital signs (heart rate, body temperature, etc.)
• Blood tests and other laboratory tests
• Disease severity assessments
• Imaging
• Medications and treatment history
• Doctor’s notes
  

If you are a participant in the olipudase alfa clinical trials ASCEND (NCT02004691), ASCEND-Peds (NCT02292654), or Long-Term Safety 13632 (NCT02004704), the following information will be collected:

• Name and contact information of the Principal Investigator
• Your participant ID in the trial
• Date that you enrolled in the trial
  

There is no direct medical benefit to you from being in this study.  The information gained from this study may help scientists and doctors to learn more about the health of people who are diagnosed with ASMD. You and other people with ASMD may benefit from results of this research in the future.

You will receive 1 year of free access to PicnicHealth’s medical record collection and management services starting from the day that you enroll in this study. As part of these services, PicnicHealth will:

• Collect your medical records on your behalf from the healthcare providers that you authorize
• Digitize your medical records so that you can access all of them online through your PicnicHealth account
• Enable you to electronically share your medical records with your doctors, care givers, and family
  

To learn more about PicnicHealth’s services, you can visit https://picnichealth.com/SG-ASMD.

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You can choose not to participate. Your medical data will not be used, and you will not receive PicnicHealth’s medical record collection service free of charge.  This will not affect in any way the regular care you receive from your doctors.

You will not be paid for taking part in this study. You will receive PicnicHealth’s medical record collection services free of charge for 1 year. PicnicHealth will collect and digitize your medical records and make the records available to you through the PicnicHealth timeline. After 1 year, if you would like PicnicHealth to continue collecting your new medical records on an ongoing basis, you will have the choice to pay for PicnicHealth’s services. If you choose not to pay for this service, you can continue to access your existing medical records through your PicnicHealth account, and no new records will be added. You can request that your data be deleted from PicnicHealth at any time, but your data cannot be deleted from the study once it has been collected.

There are no physical risks from participating in this study. Many precautions will be taken to protect your information. PicnicHealth has a series of safeguards in place, including removing PII from any transcripts, performing staff training via standard operating procedures (SOPs) to ensure that every precaution is taken, identifying study documents by number only, and so forth. Despite best efforts, it is not possible to guarantee that your personally identifiable information (e.g., name, date of birth) will never become known. For example, data may be vulnerable in transit or servers may be accessed by unauthorized individuals. However, the risk for such a confidentiality breach is low. Your private health information will be kept secure as below.

You will be assigned a unique patient identification number.  The medical data used for this study will be labeled with this identification number; the data will not be labeled with your name, picture, or any other personally identifying information.  The de-identified data will be kept in data warehouses within Sanofi Genzyme. No person will be identified in any report or publication from this study.

National applicable laws and regulations to protect the personalized data in your medical records will be strictly followed.

People that may look at and/or copy your medical records for research, quality assurance, and data analysis include:

• The investigator
• PicnicHealth personnel directly involved with the medical data retrieval processes
• The Institutional Review Board responsible for protecting the rights and safety of the patients who take part in research studies
• Regulatory health authorities (government agencies involved in keeping research safe for people)
  

The Institutional Review Board (IRB), IntegReview, and accrediting agencies may inspect and copy your records, which may have your name on them.  Therefore, total confidentiality cannot be guaranteed.  If the study results are presented at meetings or printed in publications, your name will not be used.

If you sign this consent form, you give permission to PicnicHealth to use or disclose the health information in your medical records only for the purposes of this research study.  No names or personally identifying information will be disclosed to Sanofi Genzyme. Your authorization to use and disclose your health information does not have an expiration date, but that usage will only be for the purposes described in this consent form.

Health information that identifies you may be disclosed to the Institutional Review Board, and representatives of the United States Food and Drug Administration (FDA) or other national and local health authorities.

You may change your mind and revoke (take back) this authorization at any time, without penalty or other consequence.  If you revoke this authorization, no additional health information will be retrieved about you.  However, any data that has already been retrieved, up until the time you notify PicnicHealth to revoke your authorization, will remain accessible to Sanofi Genzyme.  These data will not include your name or personally identifying information.

Any publications resulting from this research will be shared with you.

You can contact PicnicHealth for any questions.

Email: SG-ASMD@picnichealth.com

Phone: (415) 801-0572

If you do not want to talk to PicnicHealth, or if you have concerns or complaints about the research, or if you want to ask about your rights, you may contact IntegReview.  IntegReview is a group of people that has reviewed this research study.  The main goal of this review is to protect the rights and well-being of the human subjects participating in research studies.  IntegReview’s policy indicates that all concerns/complaints are to be submitted in writing for review to:

Mailing Address:

‍Chairperson

IntegReview IRB

3815 S. Capital of Texas Highway

Suite 320

Austin, Texas 78704

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‍Email:

integreview@integreview.com

If you are unable to provide your concerns/complaints in writing or if this is an emergency situation regarding subject safety, contact IntegReview’s office at:

Phone:

512-326-3001

toll free at 1-877-562-1589

(between 8 am and 5 pm Central Time)

IntegReview has approved the information in this consent form and has given approval for the investigator to do the study. This does not mean IntegReview has approved your being in the study. You must consider the information in this consent form for yourself and decide if you want to be in this study.

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It is your choice if you want to be in the study. No one can force you to be in the study. You may not want to be in this study or you may leave the study at any time without penalty or loss of benefits to which you are otherwise entitled.

There are no additional costs to be in this study. You are still responsible for the costs of medical care and treatment for your disease.

You will not lose any of your legal rights by signing this consent form.

If there is new information or any significant new findings that could relate to your willingness to continue participation, we will tell you. You can then decide if you still want to be in the study.

You will be given a separate copy of the California Experimental Research Subject’s Bill of Rights. If you have not received a copy of this document, please notify study staff.

What is a consent form?

The informed consent document contains information required by federal regulations. The informed consent document must be approved by an Institutional Review Board (IRB).

What is an Institutional Review Board (IRB)?

An Institutional Review Board (IRB) is a group of people that reviews research studies. The main goal of this review is to protect the rights and well-being of the human subjects participating in research studies.

IntegReview, the IRB for this study:

IntegReview is an IRB whose board members provide IRB services across the United States, Latin America and Japan.

To meet requirements of the law, the IntegReview Boards currently include:

• Doctors
• Pharmacists
• Nurses
• Toxicologists (people who study the harmful effects of chemicals)
• Other specialists
• Others who do not have a background in science/medicine