Cystic FibrosisGOAL- e2NCT01521338
G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)
An observational study of Cystic Fibrosis, run by Steven M Rowe at 36 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 120 participants
- Where
- 36 sitesUnited States
- Study dates
- Jan 2014to Jun 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Cystic Fibrosis and the G551D, R117H and non-G551D gating Mutations ages 6 years old and older
Who may be able to join
- Male or female ≥ 6 years of age at Visit 1. :
- Must have a clinical diagnosis of cystic fibrosis and the following CFTR mutations:
- For Cohort 1 (Closed to enrollment June 30, 2012):
- G551D on at least 1 allele Any known or unknown mutations allowed on second allele.
- For Cohort 2:
- R117H on at least 1 allele Any known or unknown mutation on the second allele except G551D
Show all 11Show fewer
- 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
- 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.)
- Clinically stable with no significant changes in health status within the 14 days prior to Visit 1.
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
Who cannot join
- 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.
- Any upper or lower respiratory symptoms requiring treatment with oral, inhaled or IV antibiotics within the 2 weeks prior to Visit 1.
- History of solid organ transplantation.
- 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.
Trial locations and contacts
36 sites in 1 country.
36 sites in 1 country
United States36 sites
University of Michigan
Ann Arbor, Michigan
The Children's Hospital Colarado
Aurora, Colorado, 80045
The Johns Hopkins University
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Massachusetts General Hospital
Boston, Massachusetts, 02114
Children's Hospital Boston
Boston, Massachusetts, 02115
Women and Children's Hospital of Buffalo
Buffalo, New York, 14222
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29403
University of Chicago
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
Nation Wide Childrens Hospital
Columbus, Ohio
University of Texas Southwestern/Children's Medical Center of Dallas
Dallas, Texas, 75390-8558
National Jewish Health
Denver, Colorado, 80206
Children's Hospital of Michigan
Detroit, Michigan, 48201
Grand Rapids CF Center
Grand Rapids, Michigan, 49503
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children Indiana University Medical Center
Indianapolis, Indiana, 46202
Indianapolis University
Indianapolis, Indiana
The Children's Mercy Hospital--University of Missouri at Kansas City
Kansas City, Missouri, 64108
Dartmouth Hitchcock Clinic-Lebanon
Lebanon, New Hampshire
University of Kentucky
Lexington, Kentucky
University of Arkansas for Medical Sciences
Little Rock, Arkansas
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin
University of Minnesota
Minneapolis, Minnesota, 55455
West Virginia University CF Medical Center
Morgantown, West Virginia, 26506
Vanderbilt Children's Hospital Vanderbilt University Medical Center
Nashville, Tennessee, 37232-9500
Yale
New Haven, Connecticut
Stanford University Medical Center
Palo Alto, California, 94394
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
Phoenix Childrens Hospital
Phoenix, Arizona
Children's Hospital of Pittsburgh of UPMC- University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Oregon Health Sciences University
Portland, Oregon, 97239
Intermountain Cystic Fibrosis Center University of Utah Health Sciences Center
Salt Lake City, Utah, 84132
Toledo Children's Hospital
Toledo, Ohio
About this study
Official title: G551D Observational Study (GOAL)-Expanded to Additional Genotypes and Extended for Long Term 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.
Read the 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
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, G551D, G551D Mutation
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Endpoint for the Core Study
Change in FEV1% predicted between Visit 1 and visit 5
Measured: Change in FEV1% predicted between Visit 1 and Visit 5
Secondary outcomes (2)
Change in sweat chloride between Visit 1 and Visit 5.
Change in sweat chloride between Visit 1 and Visit 5.
Measured: VISIT 1 AND VISIT 5
Change in body weight between Visit 1 and Visit 5.
Change in body weight between Visit 1 and Visit 5.
Measured: VISIT 1 AND VISIT 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2012 (estimated)
- Study startJan 2014
- Primary completionJun 2021
- Study completionJun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Steven M Rowe
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Steven M Rowe, University of Alabama at Birmingham
- Investigators
- Steven M Rowe, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01521338
- Sponsor's protocol ID
- F11120200
- Acronym
- GOAL- e2
- Status last verified
- Jun 2021
- First submitted
- 25 Jan 2012
- Last updated
- 30 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
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):e122695. doi: 10.1172/jci.insight.122695.
PubMed 30568035van de Peppel IP, Doktorova M, Berkers G, de Jonge HR, Houwen RHJ, Verkade HJ, Jonker JW, Bodewes FAJA. IVACAFTOR restores FGF19 regulated bile acid homeostasis in cystic fibrosis patients with an S1251N or a G551D gating mutation. J Cyst Fibros. 2019 Mar;18(2):286-293. doi: 10.1016/j.jcf.2018.09.001. Epub 2018 Sep 29.
PubMed 30279125
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- G551D Observational Study (GOAL)-Expanded to Additional Genotypes and Extended for Long Term Follow up (GOAL-e2)
- ClinicalTrials.gov ID
- NCT01521338
- Acronym / sponsor ID
- GOAL- e2 / F11120200
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, G551D, G551D Mutation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Steven M RoweUniversity, hospital or other
- Study start
- Jan 2014 (actual)
- Primary completion
- Jun 2021 (actual)
- Study completion
- Jun 2021 (actual)
- First posted
- 30 Jan 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Cystic Fibrosis and the G551D, R117H and non-G551D gating Mutations ages 6 years old and older
Key inclusion criteria (11 listed)
- Male or female ≥ 6 years of age at Visit 1. :
- Must have a clinical diagnosis of cystic fibrosis and the following CFTR mutations:
- G551D on at least 1 allele Any known or unknown mutations allowed on second allele.
- R117H on at least 1 allele Any known or unknown mutation on the second allele except G551D
- 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
Key exclusion criteria (4 listed)
- 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.
- Any upper or lower respiratory symptoms requiring treatment with oral, inhaled or IV antibiotics within the 2 weeks prior to Visit 1.
- History of solid organ transplantation.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
2 secondary outcomes
Change in sweat chloride between Visit 1 and Visit 5.
Time frame: VISIT 1 AND VISIT 5
Change in body weight between Visit 1 and Visit 5.
Time frame: VISIT 1 AND VISIT 5
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jun 2021.
- Derived from this study: PubMed 30568035Donaldson 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):e122695. doi: 10.1172/jci.insight.122695.
- Derived from this study: PubMed 30279125van de Peppel IP, Doktorova M, Berkers G, de Jonge HR, Houwen RHJ, Verkade HJ, Jonker JW, Bodewes FAJA. IVACAFTOR restores FGF19 regulated bile acid homeostasis in cystic fibrosis patients with an S1251N or a G551D gating mutation. J Cyst Fibros. 2019 Mar;18(2):286-293. doi: 10.1016/j.jcf.2018.09.001. Epub 2018 Sep 29.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven M Rowe, MD principal investigator
University of Alabama at Birmingham
36 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Steven M Rowe at 36 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Endpoint for the Core Study (time frame: Change in FEV1% predicted between Visit 1 and Visit 5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01521338, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2021.