Trial Outcomes & Findings for Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (NCT NCT03801993)
NCT ID: NCT03801993
Last Updated: 2024-10-15
Results Overview
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.
Recruitment status
COMPLETED
Target enrollment
402 participants
Primary outcome timeframe
1 month
Results posted on
2024-10-15
Participant Flow
Participant milestones
| Measure |
All Enrolled
Eligible participant who fully completed at least one ePRO at Baseline Visit
|
|---|---|
|
Overall Study
STARTED
|
402
|
|
Overall Study
Week 1
|
329
|
|
Overall Study
Week 2
|
324
|
|
Overall Study
Week 4
|
312
|
|
Overall Study
COMPLETED
|
254
|
|
Overall Study
NOT COMPLETED
|
148
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Age <18
n=169 Participants
Age \<18 years at baseline among All Enrolled
|
Age >=18
n=233 Participants
Age \>= 18 years at baseline among All Enrolled
|
Total
n=402 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
169 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
169 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
233 Participants
n=7 Participants
|
233 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
10.28 years
n=5 Participants
|
26.23 years
n=7 Participants
|
19.36 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
75 Participants
n=5 Participants
|
118 Participants
n=7 Participants
|
193 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
94 Participants
n=5 Participants
|
115 Participants
n=7 Participants
|
209 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
157 Participants
n=5 Participants
|
226 Participants
n=7 Participants
|
383 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
164 Participants
n=5 Participants
|
218 Participants
n=7 Participants
|
382 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Genotype
F508 Heterozygous
|
64 Participants
n=5 Participants
|
78 Participants
n=7 Participants
|
142 Participants
n=5 Participants
|
|
Genotype
F508 Homozygous
|
89 Participants
n=5 Participants
|
126 Participants
n=7 Participants
|
215 Participants
n=5 Participants
|
|
Genotype
Other/Unknown
|
16 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Mutation Class
I-III (more severe)
|
129 Participants
n=5 Participants
|
185 Participants
n=7 Participants
|
314 Participants
n=5 Participants
|
|
Mutation Class
IV-V (less severe)
|
13 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Mutation Class
Unknown
|
27 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
58 Participants
n=5 Participants
|
|
Sweat Chloride at Diagnosis
|
91.00 mEq/L
STANDARD_DEVIATION 22.08 • n=5 Participants
|
97.53 mEq/L
STANDARD_DEVIATION 18.05 • n=7 Participants
|
94.73 mEq/L
STANDARD_DEVIATION 20.11 • n=5 Participants
|
|
Pancreatic Sufficiency
Insufficient
|
149 Participants
n=5 Participants
|
204 Participants
n=7 Participants
|
353 Participants
n=5 Participants
|
|
Pancreatic Sufficiency
Sufficient
|
20 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
49 Participants
n=5 Participants
|
|
Modulator Use
None
|
87 Participants
n=5 Participants
|
87 Participants
n=7 Participants
|
174 Participants
n=5 Participants
|
|
Modulator Use
Ivacaftor
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Modulator Use
Lumacaftor/Ivacaftor
|
55 Participants
n=5 Participants
|
38 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Modulator Use
Tezacaftor/Ivacaftor
|
16 Participants
n=5 Participants
|
87 Participants
n=7 Participants
|
103 Participants
n=5 Participants
|
|
Modulator Use
Triple Combination
|
1 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Modulator Use
Ivacaftor and Triple Combination
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 monthNumber 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.
Outcome measures
| Measure |
Age <18
n=169 Participants
Age \<18 years at baseline among All Enrolled
|
Age >=18
n=233 Participants
Age \>= 18 years at baseline among All Enrolled
|
|---|---|---|
|
Number of Participants Who Fully Completed at Least One ePRO
Week 1
|
133 Participants
|
196 Participants
|
|
Number of Participants Who Fully Completed at Least One ePRO
Week 2
|
130 Participants
|
194 Participants
|
|
Number of Participants Who Fully Completed at Least One ePRO
Week 4
|
125 Participants
|
187 Participants
|
SECONDARY outcome
Timeframe: 1 monthPopulation: Participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks
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.
Outcome measures
| Measure |
Age <18
n=95 Participants
Age \<18 years at baseline among All Enrolled
|
Age >=18
n=151 Participants
Age \>= 18 years at baseline among All Enrolled
|
|---|---|---|
|
Number of Participants With Period-Prevalence of Constipation
|
18 Participants
|
35 Participants
|
SECONDARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Age <18
n=169 Participants
Age \<18 years at baseline among All Enrolled
|
Age >=18
n=233 Participants
Age \>= 18 years at baseline among All Enrolled
|
|---|---|---|
|
Mean Patient Reported Outcome (PRO) Scores
PAC-SYM Total Score
|
0.49 score on a scale
Standard Deviation 0.45
|
0.64 score on a scale
Standard Deviation 0.55
|
|
Mean Patient Reported Outcome (PRO) Scores
PAGI-SYM Total Score
|
0.49 score on a scale
Standard Deviation 0.56
|
0.78 score on a scale
Standard Deviation 0.66
|
|
Mean Patient Reported Outcome (PRO) Scores
PAC-QOL Total Score
|
0.58 score on a scale
Standard Deviation 0.47
|
0.73 score on a scale
Standard Deviation 0.59
|
SECONDARY outcome
Timeframe: At Visit 1 (1 day)Number of enrolled participants receiving treatment for GI symptoms at Visit 1
Outcome measures
| Measure |
Age <18
n=169 Participants
Age \<18 years at baseline among All Enrolled
|
Age >=18
n=233 Participants
Age \>= 18 years at baseline among All Enrolled
|
|---|---|---|
|
Number of Participants Treated for GI Symptoms
Initiating or Receiving GI Treatment
|
167 Participants
|
228 Participants
|
|
Number of Participants Treated for GI Symptoms
GI Treatment for Constipation
|
88 Participants
|
78 Participants
|
|
Number of Participants Treated for GI Symptoms
GI Treatment for Non-Constipation
|
166 Participants
|
228 Participants
|
|
Number of Participants Treated for GI Symptoms
Use GI Tube
|
24 Participants
|
8 Participants
|
|
Number of Participants Treated for GI Symptoms
On Azithromycin
|
60 Participants
|
131 Participants
|
|
Number of Participants Treated for GI Symptoms
On drugs used in diabetes
|
12 Participants
|
59 Participants
|
Adverse Events
All Subjects
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Dr. Christopher H. Goss M.D., M.Sc.
Seattle Children's Hospital
Phone: (206) 987-5725
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place