Cystic FibrosisGALAXYNCT03801993
Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
An observational study of Cystic Fibrosis, run by Chris Goss at 26 sites in United States.
Trial summary
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 402 participants
- Where
- 26 sitesUnited States
- Study dates
- 1 May 2019to 17 Sep 2019
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.
Who may be able to join
- All genders ≥ 2 years of age at time of consent
- Documentation of a Cystic Fibrosis (CF) diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
- Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
- Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproterenol of less than -5 mV)
- Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled)
Show all 9Show fewer
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
- Willing to complete questionnaires on mobile device
- Able to use the Medidata Patient Cloud mobile application for completing the questionnaires
Who cannot join
- Presence of a condition or abnormality that, in the opinion of the Investigator, would complicate interpretation of study outcome data or interfere with achieving the study objectives
- Presence of a pulmonary exacerbation at the Enrollment Visit
- Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit
- Current gastrointestinal (GI) or abdominal/pelvic malignancy
- Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit
- At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel cleanout in the 28 days after the Enrollment Visit
- Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment Visit
- Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
United States26 sites
Children's Healthcare of Atlanta and Emory University
Atlanta, Georgia, 30322
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, 02115
The Children's Specialty Center Fletcher Allen Health Care
Burlington, Vermont, 05401
Atrium Health Pulmonary Care
Charlotte, North Carolina, 28203
Northwestern University
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern / Children's Health
Dallas, Texas, 75247
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Central Connecticut Cystic Fibrosis Center
Hartford, Connecticut, 06106
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Kentucky
Lexington, Kentucky, 40506-9983
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08903
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, 73104
Stanford University Medical Center
Palo Alto, California, 94304
Nemours Children's Clinic - Pensacola
Pensacola, Florida, 32514
OSF Saint Francis Medical Center
Peoria, Illinois, 61637
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Maine Medical Center
Portland, Maine, 04102
Oregon Health Sciences University
Portland, Oregon, 97239
Intermountain Cystic Fibrosis Center
Salt Lake City, Utah, 84113
SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
All Children's Hospital
St. Petersburg, Florida, 33701
Children's National Medical Center
Washington D.C., District of Columbia, 20816
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, 27103
University of Massachusetts Memorial Health Care
Worcester, Massachusetts, 01655
About this study
Official title: Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
Read the detailed description
There are currently no large, multicenter prospective clinical trials examining management of constipation or other gastrointestinal (GI) symptoms in people with cystic fibrosis (CF). Current recommendations in the CF literature are largely based on expert consensus and opinions. Yet, constipation and other GI symptoms are crucial factors in quality of life (QOL) and maintenance of optimal nutritional state in people with CF. This study will use GI-symptomatology questionnaires to understand the multiple overlapping GI symptoms in people with CF.
Eligible subjects will be consented and enrolled in the study at the Enrollment Visit. At the visit, the subject or parent/guardian will complete the patient reported outcome surveys (PROs) using a mobile device (e.g., smartphone or tablet). The same questionnaires will be completed on a mobile device outside the clinic three additional times. The PROs will consist of four questionnaires: Patient Assessment of Constipation Symptoms (PAC-SYM), Patient Assessment of Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation Quality of Life (PAC-QOL) and a disease-specific questionnaire (Bristol Stool Scale and questions about fecal incontinence, and stool quality and frequency).
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CF, GI, nutrition, quality of life, bowel, constipation, gastrointestinal
- Medical subject headings
- Cystic Fibrosis, Constipation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| All Subjects | Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Fully Completed at Least One ePRO
Number participants with scheduled outside-the-clinic assessments who fully completed at least one of the four PROs. A "fully complete" questionnaire is defined as having responded to all questions with no missing responses.
Measured: 1 month
Secondary outcomes (3)
Number of Participants With Period-Prevalence of Constipation
This table summarizes the period-prevalence of protocol-defined constipation. For each follow-up response, constipation was defined by the protocol as having fewer than 3 bowel movements and/or Bristol Stool Scale of Type 1 or 2 (hard lumps) in the past 7 days. Period prevalence is defined as occurrence of the symptom at any time from baseline to week 4. Only participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.
Measured: 1 month
Mean Patient Reported Outcome (PRO) Scores
Mean PRO scores (PAC-SYM score range: 0-4, PAGI-SYM score range: 0-5, PAC-QOL score range: 0-4) at time of enrollment (Visit 1) where lower scores correspond to less symptom severity.
Measured: At Visit 1 (1 day)
Number of Participants Treated for GI Symptoms
Number of enrolled participants receiving treatment for GI symptoms at Visit 1
Measured: At Visit 1 (1 day)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2019
- Study start1 May 2019
- Primary completion17 Sep 2019
- Study completion17 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)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
- 402 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chris Goss
- Sponsor type
- University, hospital or other
- Collaborators
- Emory University, University of North Carolina, Charlotte, University of Texas, Cystic Fibrosis Foundation
- Responsible party
- Sponsor-investigator, Chris Goss, Seattle Children's Hospital
- Investigators
- A. Jay Freeman, MD, MSc, Principal investigator, Emory University/Children's HealthcareBaharak Moshiree, MD, Principal investigator, University of North Carolina Charlotte, Atrium HealthMeghana Sathe, MD, Principal investigator, University of Texas Southwestern/Children's Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03801993
- Sponsor's protocol ID
- GALAXY-OB-18
- Acronym
- GALAXY
- Status last verified
- Oct 2024
- First submitted
- 4 Jan 2019
- Last updated
- 15 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Moshiree B, Freeman AJ, Vu PT, Khan U, Ufret-Vincenty C, Heltshe SL, Goss CH, Schwarzenberg SJ, Freedman SD, Borowitz D, Sathe M; GALAXY Study Group. Multicenter prospective study showing a high gastrointestinal symptom burden in cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):266-274. doi: 10.1016/j.jcf.2022.10.006. Epub 2022 Oct 29.
PubMed 36319569
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)
- ClinicalTrials.gov ID
- NCT03801993
- Acronym / sponsor ID
- GALAXY / GALAXY-OB-18
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CF, GI, nutrition, quality of life, bowel, constipation, gastrointestinal
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 402 participants (actual)
- Lead sponsor
- Chris GossUniversity, hospital or other
- Collaborators
- Emory University, University of North Carolina, Charlotte, University of Texas, Cystic Fibrosis Foundation
- Study start
- 1 May 2019 (actual)
- Primary completion
- 17 Sep 2019 (actual)
- Study completion
- 17 Sep 2019 (actual)
- First posted
- 14 Jan 2019 (actual)
- Results
- Posted 15 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
Detailed description
There are currently no large, multicenter prospective clinical trials examining management of constipation or other gastrointestinal (GI) symptoms in people with cystic fibrosis (CF). Current recommendations in the CF literature are largely based on expert consensus and opinions. Yet, constipation and other GI symptoms are crucial factors in quality of life (QOL) and maintenance of optimal nutritional state in people with CF. This study will use GI-symptomatology questionnaires to understand the multiple overlapping GI symptoms in people with CF.
Eligible subjects will be consented and enrolled in the study at the Enrollment Visit. At the visit, the subject or parent/guardian will complete the patient reported outcome surveys (PROs) using a mobile device (e.g., smartphone or tablet). The same questionnaires will be completed on a mobile device outside the clinic three additional times. The PROs will consist of four questionnaires: Patient Assessment of Constipation Symptoms (PAC-SYM), Patient Assessment of Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation Quality of Life (PAC-QOL) and a disease-specific questionnaire (Bristol Stool Scale and questions about fecal incontinence, and stool quality and frequency).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Subjects | – | –Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.
Key inclusion criteria (9 listed)
- All genders ≥ 2 years of age at time of consent
- Documentation of a Cystic Fibrosis (CF) diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled)
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
- Willing to complete questionnaires on mobile device
Key exclusion criteria (8 listed)
- Presence of a condition or abnormality that, in the opinion of the Investigator, would complicate interpretation of study outcome data or interfere with achieving the study objectives
- Presence of a pulmonary exacerbation at the Enrollment Visit
- Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit
- Current gastrointestinal (GI) or abdominal/pelvic malignancy
- Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit
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
Number of Participants Who Fully Completed at Least One ePRO
Time frame: 1 month
Number participants with scheduled outside-the-clinic assessments who fully completed at least one of the four PROs. A "fully complete" questionnaire is defined as having responded to all questions with no missing responses.
3 secondary outcomes
Number of Participants With Period-Prevalence of Constipation
Time frame: 1 month
Mean Patient Reported Outcome (PRO) Scores
Time frame: At Visit 1 (1 day)
Number of Participants Treated for GI Symptoms
Time frame: At Visit 1 (1 day)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36319569Moshiree B, Freeman AJ, Vu PT, Khan U, Ufret-Vincenty C, Heltshe SL, Goss CH, Schwarzenberg SJ, Freedman SD, Borowitz D, Sathe M; GALAXY Study Group. Multicenter prospective study showing a high gastrointestinal symptom burden in cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):266-274. doi: 10.1016/j.jcf.2022.10.006. Epub 2022 Oct 29.
- Individual participant dataSharing planned: No
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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.
A. Jay Freeman, MD, MSc principal investigator
Emory University/Children's Healthcare
Baharak Moshiree, MD principal investigator
University of North Carolina Charlotte, Atrium Health
Meghana Sathe, MD principal investigator
University of Texas Southwestern/Children's Health
26 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 Chris Goss at 26 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 9 inclusion and 8 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 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 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?
Yes. Results are posted in the registry (first posted 15 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants Who Fully Completed at Least One ePRO (time frame: 1 month). 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 NCT03801993, which also lists 1 linked publication.
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 15 Oct 2024.