CHOP IRB#: «ID»

Effective Date: «ApprovalDate»

Expiration Date: «ExpirationDate» Page 1 of 10

Informed Consent Form and HIPAA Authorization

Study Title: A randomized, placebo-controlled trial of NewCure® for the treatment of rare disease in children

Version Date: May 6, 2014

You, or your child, may be eligible to take part in a research study. This form gives you

important information about the study. It describes the purpose of this research study, and

the risks and possible benefits of participating.

If there is anything in this form you do not understand, please ask questions. Please take

your time. You do not have to take part in this study if you do not want to. If you take part, you can leave the study at any time.

In the sections that follow, the word “we” means the study doctor and other research staff. If you are a parent or legal guardian who is giving permission for a child, please

note that the word “you” refers to your child.

Why are you being asked to take part in this study?

You are being invited to take part in this research study because you have a rare disease

and it has not responded to standard treatment.

What is the purpose of this research study?

The purpose of this research study is to determine if NewDrug is safe and effective for

the treatment of rare disease.

Children with rare disease don’t make a normal amount of a chemical called SEAN.

· Gromit is vital for the digestion of milk products such as cheese.

· In mice and in adult volunteers NewDrug resulted in a normal digestion of cheese.

· It is not known whether NewDrug will work when given to children.

· NewDrug is not yet approved by the US Food and Drug Administration and is

available only to participants in this research study.

How many people will take part?

About 100 children at 10 medical centers will take part in the study, including

approximately 10 participants from CHOP.

Principal Investigator: Dr. Wallace Telephone: (215) 590-1000

Division of Gastroenterology

Emergency Contact: Mr. Gromit, RN Telephone: (215) 590-1001

Only the PI needs to be

listed as an investigator

Emergency contact must connect to a real

person. Not needed for minimal risk studies

KEY

Elements of Consent (§46.116) and HIPAA (§164.508)

(a)(1 - 8) = basic elements of consent

(b)(1-6) = additional elements of consent

(c)(1)(i-vi) = core elements of HIPAA authorization

(c)(2)(i-iii) = required HIPAA statements

(a)(1)

(a)(8)

(a)(1)

* Text column limited to 6 inches.

* Written in 2nd person

• Bullet points instead of dense paragraphs

* White space between paragraphs

Question & answer format

(b)(6)

CHOP IRB#: «ID»

Effective Date: «ApprovalDate»

Expiration Date: «ExpirationDate» Page 2 of 10

What is involved in the study?

If you agree to take part, your participation will last for 3 months. The study involves 5

clinic visits.

Study Procedures

If you take part in this study you will have the following tests and procedures. Some of

the procedures may be repeated several times. Tests that are part of your regular, routine

medical care will continue to be performed. Additional tests may be performed if any of

your initial test results are not normal.

Interviews: A team member will take your medical history, along with a listing of any

medications you are taking. Throughout the study you will be asked to

report if you think that anything bad has happened as a result of the study.

Physical Examination: Exams will be conducted before and during the study including

measurements of weight, height, blood pressure, heart rate and respiratory

rate, etc.

Pregnancy Test: If you are eleven years old or older or have already started having

periods, you will be asked to take a urine pregnancy test before starting

this study. The results will be shared with you and not with your parent(s).

We strongly encourage you to share the results with your parents. If you

are found to be pregnant, you will not be able to continue participation in

the study.

Blood Tests: Blood tests will be taken to measure your blood count and blood

chemistries. These tests will be repeated several times. Approximately 1 –

2 teaspoons of blood will be needed each time, depending on the visit.

Study Medication: You will need to take the study medication twice a day by mouth

for 4 weeks.

Depending on which group you are in, your study medication will be

either a placebo (an inactive pill, like a sugar pill), a low dose of New

Drug or a high dose of NewDrug. Some subjects will take placebo in order

to help determine if NewDrug makes a difference.

You will be assigned to a group by a process called randomization.

Randomization is similar to drawing lots or flipping a coin. Neither you

nor your study doctor will know which group you are in. This allows for

an honest assessment of your response.

Questionnaires: You will complete a survey about how you feel after eating cheese

before the study begins and after 4 weeks on study medication.

(a)(1)

• These fields are used by the IRB Office to "stamp" the ICF.

• To modify the consent form, use the most recent approved

“clean” version in eIRB Section 12.01 (2.0)

(c)(1)(i)

Procedures should be explained only once