Trial Outcomes & Findings for Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations (NCT NCT03624101)
NCT ID: NCT03624101
Last Updated: 2025-04-16
Results Overview
Percent change in FEV1 from Baseline and 24 weeks
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
1 participants
Primary outcome timeframe
24 weeks
Results posted on
2025-04-16
Participant Flow
Participant milestones
| Measure |
Symdeko
If the participant is not on a current modulator, they will take Symdeko for 28 days followed by a 28 day off period. This cycle will be continued for 168 days
Symdeko: CFTR modulator
|
Symdeko/Ivacaftor
If the participant currently takes Symdeko and Ivacaftor, they will take Symdeko for a 28 day period followed by Ivacaftor for a 28 day period. This cycle will be continued for 168 days
symdeko/Ivacaftor: CFTR modulator
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
0
|
|
Overall Study
COMPLETED
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations
Baseline characteristics by cohort
| Measure |
Symdeko
n=1 Participants
If the participant is on symdeko, they will take symdeko for 28 days followed by a 28 day off period. This cycle will be continued for 168 day
Symdeko: CFTR modulator
|
Symdeko/Ivacaftor
If the participant currently takes Symdeko , they will take Symdeko for a 28 day period followed by Ivacaftor for a 28 day period. This cycle will be continued for 168 days
symdeko/Ivacaftor: CFTR modulator
|
Total
n=1 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=39 Participants
|
—
|
0 Participants
n=35 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=39 Participants
|
—
|
1 Participants
n=35 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
—
|
0 Participants
n=35 Participants
|
|
Age, Continuous
|
37 years
n=39 Participants
|
—
|
37 years
n=35 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=39 Participants
|
—
|
0 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=39 Participants
|
—
|
1 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=39 Participants
|
—
|
1 participants
n=35 Participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: no improvement was noticed and the study ended prematurely. No formal statistical analysis was performed
Percent change in FEV1 from Baseline and 24 weeks
Outcome measures
| Measure |
Symdeko
n=1 Participants
If the participant is not on a current modulator, they will take Symdeko for 28 days followed by a 28 day off period. This cycle will be continued for 168 days
Symdeko: CFTR modulator
|
Symdeko/Ivacaftor
If the participant currently takes Symdeko, they will take Symdeko for a 28 day period followed by Ivacaftor for a 28 day period. This cycle will be continued for 168 days
symdeko/Ivacaftor: CFTR modulator
|
|---|---|---|
|
Percent Change in FEV1 From Baseline
|
0 change in percentage
|
—
|
Adverse Events
Symdeko
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Symdeko/Ivacaftor
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Symdeko
n=1 participants at risk
If the participant is not on a current modulator, they will take Symdeko for 28 days followed by a 28 day off period. This cycle will be continued for 168 days
Symdeko: CFTR modulator
|
Symdeko/Ivacaftor
If the participant currently takes Symdeko , they will take Symdeko for a 28 day period followed by Ivacaftor for a 28 day period. This cycle will be continued for 168 days
symdeko/Ivacaftor: CFTR modulator
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Increased cough
|
100.0%
1/1 • Number of events 2 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
—
0/0 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
|
Respiratory, thoracic and mediastinal disorders
increased sputum production
|
100.0%
1/1 • Number of events 2 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
—
0/0 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
|
General disorders
Increased fatigue
|
100.0%
1/1 • Number of events 2 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
—
0/0 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
|
Metabolism and nutrition disorders
decreased appetite
|
100.0%
1/1 • Number of events 1 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
—
0/0 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
|
Endocrine disorders
low blood glucose
|
100.0%
1/1 • Number of events 1 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
—
0/0 • 6 months
only 1 patient was entered into the study (Symdeko Arm). No patients were entered into the Symdeko/Ivacaftor Arm
|
Additional Information
Heather Hathorne
University of Alabama at Birmingham Child Health Research Unit
Phone: 2055032829
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place