Trial Outcomes & Findings for A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC) (NCT NCT00434278)
NCT ID: NCT00434278
Last Updated: 2017-05-16
Results Overview
Change in distance walked was defined as (distance walked in 6 minutes at baseline \[Day 0\]) - (distance walked in 6 minutes at Day 14) in meters.
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
27 participants
Primary outcome timeframe
From baseline to Day 14
Results posted on
2017-05-16
Participant Flow
Participant milestones
| Measure |
Placebo
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
13
|
|
Overall Study
COMPLETED
|
12
|
13
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
Baseline characteristics by cohort
| Measure |
Placebo
n=14 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
n=13 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
24.6 years
STANDARD_DEVIATION 7.0 • n=5 Participants
|
29.8 years
STANDARD_DEVIATION 10.0 • n=7 Participants
|
27.1 years
STANDARD_DEVIATION 8.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From baseline to Day 14Population: Randomized patients. For placebo arm: 2 placebo patients who were randomized did not complete the study and therefore had no values to calculate change from baseline computation.
Change in distance walked was defined as (distance walked in 6 minutes at baseline \[Day 0\]) - (distance walked in 6 minutes at Day 14) in meters.
Outcome measures
| Measure |
Placebo
n=12 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
n=13 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
|---|---|---|
|
Change in Distance Walked in the 6-minute Walk Test
|
25.2 Meters
Standard Deviation 79.8
|
26.9 Meters
Standard Deviation 60.0
|
SECONDARY outcome
Timeframe: From baseline to Day 14Population: Randomized patients. Patients not included in this analysis did not meet ATS reproducibility criteria. Two patients in the placebo arm whose screening visit values did not meet ATS reproducibility criteria were randomized in error and completed the study.
FEV1 (forced expiratory volume in 1 second) and FVC (forced vital capacity) were recorded at Day 0 (baseline) and Day 14. Change in FEV1 and FVC from baseline to Day 14 was reported as a percentage of values predicted for age, height, and race.
Outcome measures
| Measure |
Placebo
n=11 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
n=12 Participants
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
|---|---|---|
|
Change in Pulmonary Function as Measured by FEV1 and FVC
FVC
|
-1.2 Percentage of predicted value
Standard Deviation 4.5
|
1.0 Percentage of predicted value
Standard Deviation 4.8
|
|
Change in Pulmonary Function as Measured by FEV1 and FVC
FEV1
|
-0.5 Percentage of predicted value
Standard Deviation 4.1
|
0.2 Percentage of predicted value
Standard Deviation 2.8
|
Adverse Events
Placebo
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Dornase Alfa
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=14 participants at risk
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
n=13 participants at risk
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
|---|---|---|
|
Congenital, familial and genetic disorders
Cystic Fibrosis
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Congenital, familial and genetic disorders
Cystic Fibrosis Lung
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Gastrointestinal disorders
Distal Intestinal Obstruction Syndrome
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
Other adverse events
| Measure |
Placebo
n=14 participants at risk
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
Dornase Alfa
n=13 participants at risk
2.5 mg inhalation dose twice daily for 14±2 days (Visit 2 to Visit 3)
|
|---|---|---|
|
Congenital, familial and genetic disorders
Cystic Fibrosis
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
General disorders
Fatigue
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
General disorders
Chest Discomfort
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
General disorders
Non-Cardiac Chest Pain
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Nervous system disorders
Headache
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
7.1%
1/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
0.00%
0/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Productive Cough
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis Allergic
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Tract Congestion
|
0.00%
0/14
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
7.7%
1/13
Randomized (same as safety-evaluable) patients. Note: The incidence of each AE/SAE is reported as the number of participants experiencing the event, not the number of occurrences for each AE/SAE.
|
Additional Information
Medical Communications
Genentech, Inc.
Phone: 800-821-8590
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER