G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)

- Blood Collection: Blood will be collected to be kept for research in the future and to
measure the number of blood cells called neutrophils. A small needle will be used to
collect blood from a vein in your arm. About 7-9 teaspoons of blood would be collected.

- Sweat Collection: Your sweat will be collected with a special sweat collection machine
to test the amount of salt in your sweat.

- Spirometry: You will be asked to take a test that measures how well your lungs are
working. You will be asked to take a deep breath and then blow into a mouthpiece as hard
as possible and for as long as possible. This is the same test that is done when you
come to clinic.

- Urine Collection: Urine will also be collected to be kept for research in the future.
You will be asked to pee in a cup.

- Medical Information: We are asking you to share your medical information with study
researchers. Your medical information will not contain any of your personal
identification information, like your name and address.

Optional:

• Induced Sputum Collection: If you say "yes" to collecting a sputum sample, you will be
asked to breathe in a salt water solution to help you cough out sputum. If you cannot breathe
in the salt water solution to cough out sputum for the last study visit, you will be asked to
cough mucus into a cup

Inclusion Criteria for Core Study:

1. Male or female ≥ 6 years of age at Visit 1. :

2. Must have a clinical diagnosis of cystic fibrosis and the following CFTR mutations:

1. For Cohort 1 (Closed to enrollment June 30, 2012):

G551D on at least 1 allele Any known or unknown mutations allowed on second
allele.

2. For Cohort 2:

R117H on at least 1 allele Any known or unknown mutation on the second allele
except G551D

3. For Cohort 3:

A Non-G551D gating mutation on one allele: (G178R, S549N, S549R, G551S,G970R, G1244E,
S1251N, S1255P, G1349D) Any known or unknown mutation on the second allele except
G551D OR R117H

3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (with the exception of
Canadian sites). (Patients may enroll in the Registry at Visit 1 if not previously
enrolled.)

4. Clinically stable with no significant changes in health status within the 14 days
prior to Visit 1.

5. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative.

Exclusion Criteria for Core Study

1. Participation in the VX-770-105, VX-770-106, VX-770-108, VX-770-110, VX-770-111,
VX-770-112, or VX-770-113 study, VX-770 Extended Access Program or use of ivacaftor
within 6 months prior to Visit 1.

2. Any upper or lower respiratory symptoms requiring treatment with oral, inhaled or IV
antibiotics within the 2 weeks prior to Visit 1.

3. History of solid organ transplantation.

4. Presence of a condition or abnormality that in the opinion of the investigator would
compromise the safety of the patient or the quality of the data.