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

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ClinicalTrials.gov Identifier: NCT01521338
Recruitment Status : Active, not recruiting
First Posted : January 30, 2012
Last Update Posted : April 10, 2019
Sponsor:
Information provided by (Responsible Party):
Steven M Rowe, University of Alabama at Birmingham

Tracking Information
First Submitted Date January 25, 2012
First Posted Date January 30, 2012
Last Update Posted Date April 10, 2019
Study Start Date January 2014
Actual Primary Completion Date January 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 8, 2014)
Primary Endpoint for the Core Study [ Time Frame: Change in FEV1% predicted between Visit 1 and Visit 5 ]
Change in FEV1% predicted between Visit 1 and visit 5
Original Primary Outcome Measures Not Provided
Change History Complete list of historical versions of study NCT01521338 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: September 8, 2014)
  • Change in sweat chloride between Visit 1 and Visit 5. [ Time Frame: VISIT 1 AND VISIT 5 ]
    Change in sweat chloride between Visit 1 and Visit 5.
  • Change in body weight between Visit 1 and Visit 5. [ Time Frame: VISIT 1 AND VISIT 5 ]
    Change in body weight between Visit 1 and Visit 5.
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: September 8, 2014)
ASSOCIATION BETWEEN PRIMARY AND SECONDARY ENDPOINTS [ Time Frame: 1 YEAR ]
The core and sub-studies will be linked so that associations between primary and secondary endpoints from each sub-study and clinical parameters (e.g., spirometry, weight, and sweat chloride) collected as part of the Core Study may be explored.
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)
Official Title G551D Observational Study (GOAL)-Expanded to Additional Genotypes and Extended for Long Term Follow up (GOAL-e2)
Brief Summary 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.
Detailed Description
  • 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

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood, Sputum, Urine
Sampling Method Probability Sample
Study Population Patients with Cystic Fibrosis and the G551D, R117H and non-G551D gating Mutations ages 6 years old and older
Condition Cystic Fibrosis
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Donaldson SH, Laube BL, Corcoran TE, Bhambhvani P, Zeman K, Ceppe A, Zeitlin PL, Mogayzel PJ Jr, Boyle M, Locke LW, Myerburg MM, Pilewski JM, Flanagan B, Rowe SM, Bennett WD. Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR. JCI Insight. 2018 Dec 20;3(24). pii: 122695. doi: 10.1172/jci.insight.122695.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: January 27, 2012)
120
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 2020
Actual Primary Completion Date January 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01521338
Other Study ID Numbers F11120200
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Steven M Rowe, University of Alabama at Birmingham
Study Sponsor Steven M Rowe
Collaborators Not Provided
Investigators
Principal Investigator: Steven M Rowe, MD University of Alabama at Birmingham
PRS Account University of Alabama at Birmingham
Verification Date April 2019