Trial Search Results

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

The goal of this research study is to collect blood and urine samples from people who have either the R117H type of CF or the non-G551D gating type of CF to be kept for future research.We will also use some of the collected blood to measure the number of neutrophils.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Steven M Rowe

Stanford Investigator(s):


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

      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

   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.

Ages Eligible for Study

6 Years - N/A

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Angela Leung
Not Recruiting