Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, CF, GI, nutrition, quality of life, bowel, constipation, gastrointestinal
Brief summary
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).
Interventions
None listed
Sponsors
Emory University
University of North Carolina, Charlotte
University of Texas
Cystic Fibrosis Foundation
Chris Goss
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. All genders ≥ 2 years of age at time of consent 2. 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) 3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled) 4. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative 5. Willing to complete questionnaires on mobile device 6. Able to use the Medidata Patient Cloud mobile application for completing the questionnaires
Exclusion criteria
1. 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 2. Presence of a pulmonary exacerbation at the Enrollment Visit 3. Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit 4. Current gastrointestinal (GI) or abdominal/pelvic malignancy 5. Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit 6. At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel cleanout in the 28 days after the Enrollment Visit 7. Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment Visit 8. Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Fully Completed at Least One ePRO | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Period-Prevalence of Constipation | 1 month | 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. |
| Mean Patient Reported Outcome (PRO) Scores | At Visit 1 (1 day) | 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. |
| Number of Participants Treated for GI Symptoms | At Visit 1 (1 day) | Number of enrolled participants receiving treatment for GI symptoms at Visit 1 |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Age <18 Age \<18 years at baseline among All Enrolled | 169 |
| Age >=18 Age \>= 18 years at baseline among All Enrolled | 233 |
| Total | 402 |
Baseline characteristics
| Characteristic | Age <18 | Age >=18 | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 169 Participants | 0 Participants | 169 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 233 Participants | 233 Participants |
| Age, Continuous | 10.28 years | 26.23 years | 19.36 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 12 Participants | 6 Participants | 18 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 157 Participants | 226 Participants | 383 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Genotype F508 Heterozygous | 64 Participants | 78 Participants | 142 Participants |
| Genotype F508 Homozygous | 89 Participants | 126 Participants | 215 Participants |
| Genotype Other/Unknown | 16 Participants | 29 Participants | 45 Participants |
| Modulator Use Ivacaftor | 10 Participants | 10 Participants | 20 Participants |
| Modulator Use Ivacaftor and Triple Combination | 0 Participants | 1 Participants | 1 Participants |
| Modulator Use Lumacaftor/Ivacaftor | 55 Participants | 38 Participants | 93 Participants |
| Modulator Use None | 87 Participants | 87 Participants | 174 Participants |
| Modulator Use Tezacaftor/Ivacaftor | 16 Participants | 87 Participants | 103 Participants |
| Modulator Use Triple Combination | 1 Participants | 10 Participants | 11 Participants |
| Mutation Class I-III (more severe) | 129 Participants | 185 Participants | 314 Participants |
| Mutation Class IV-V (less severe) | 13 Participants | 17 Participants | 30 Participants |
| Mutation Class Unknown | 27 Participants | 31 Participants | 58 Participants |
| Pancreatic Sufficiency Insufficient | 149 Participants | 204 Participants | 353 Participants |
| Pancreatic Sufficiency Sufficient | 20 Participants | 29 Participants | 49 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 5 Participants | 6 Participants |
| Race (NIH/OMB) More than one race | 3 Participants | 4 Participants | 7 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 3 Participants | 4 Participants |
| Race (NIH/OMB) White | 164 Participants | 218 Participants | 382 Participants |
| Sex: Female, Male Female | 75 Participants | 118 Participants | 193 Participants |
| Sex: Female, Male Male | 94 Participants | 115 Participants | 209 Participants |
| Sweat Chloride at Diagnosis | 91.00 mEq/L STANDARD_DEVIATION 22.08 | 97.53 mEq/L STANDARD_DEVIATION 18.05 | 94.73 mEq/L STANDARD_DEVIATION 20.11 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
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.
Time frame: 1 month
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Age <18 | Number of Participants Who Fully Completed at Least One ePRO | Week 1 | 133 Participants |
| Age <18 | Number of Participants Who Fully Completed at Least One ePRO | Week 2 | 130 Participants |
| Age <18 | Number of Participants Who Fully Completed at Least One ePRO | Week 4 | 125 Participants |
| Age >=18 | Number of Participants Who Fully Completed at Least One ePRO | Week 1 | 196 Participants |
| Age >=18 | Number of Participants Who Fully Completed at Least One ePRO | Week 2 | 194 Participants |
| Age >=18 | Number of Participants Who Fully Completed at Least One ePRO | Week 4 | 187 Participants |
Secondary
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.
Time frame: At Visit 1 (1 day)
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Age <18 | Mean Patient Reported Outcome (PRO) Scores | PAC-SYM Total Score | 0.49 score on a scale | Standard Deviation 0.45 |
| Age <18 | Mean Patient Reported Outcome (PRO) Scores | PAGI-SYM Total Score | 0.49 score on a scale | Standard Deviation 0.56 |
| Age <18 | Mean Patient Reported Outcome (PRO) Scores | PAC-QOL Total Score | 0.58 score on a scale | Standard Deviation 0.47 |
| Age >=18 | Mean Patient Reported Outcome (PRO) Scores | PAC-SYM Total Score | 0.64 score on a scale | Standard Deviation 0.55 |
| Age >=18 | Mean Patient Reported Outcome (PRO) Scores | PAGI-SYM Total Score | 0.78 score on a scale | Standard Deviation 0.66 |
| Age >=18 | Mean Patient Reported Outcome (PRO) Scores | PAC-QOL Total Score | 0.73 score on a scale | Standard Deviation 0.59 |
Secondary
Number of Participants Treated for GI Symptoms
Number of enrolled participants receiving treatment for GI symptoms at Visit 1
Time frame: At Visit 1 (1 day)
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Age <18 | Number of Participants Treated for GI Symptoms | Initiating or Receiving GI Treatment | 167 Participants |
| Age <18 | Number of Participants Treated for GI Symptoms | GI Treatment for Constipation | 88 Participants |
| Age <18 | Number of Participants Treated for GI Symptoms | GI Treatment for Non-Constipation | 166 Participants |
| Age <18 | Number of Participants Treated for GI Symptoms | Use GI Tube | 24 Participants |
| Age <18 | Number of Participants Treated for GI Symptoms | On Azithromycin | 60 Participants |
| Age <18 | Number of Participants Treated for GI Symptoms | On drugs used in diabetes | 12 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | On Azithromycin | 131 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | Initiating or Receiving GI Treatment | 228 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | Use GI Tube | 8 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | GI Treatment for Constipation | 78 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | On drugs used in diabetes | 59 Participants |
| Age >=18 | Number of Participants Treated for GI Symptoms | GI Treatment for Non-Constipation | 228 Participants |
Secondary
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.
Time frame: 1 month
Population: Participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Age <18 | Number of Participants With Period-Prevalence of Constipation | 18 Participants |
| Age >=18 | Number of Participants With Period-Prevalence of Constipation | 35 Participants |