Trial Outcomes & Findings for Treatment of Chronic Cough in Idiopathic Pulmonary Fibrosis With Thalidomide (NCT NCT00600028)
NCT ID: NCT00600028
Last Updated: 2017-05-15
Results Overview
The primary endpoint, suppression of cough was measured by the Cough Quality of Life Questionnaire (CQLQ) to measure the effect of interventions on cough-specific quality of life. CQLQ consist of 28 questions about cough and its effects using Likert-like 4-point scales, with lower scores indicating less effect of cough on health related quality of life. CQLQ scale ranges from 28 to 112 ( The lower the value, the higher the quality of life, 28 is considered the best).
COMPLETED
PHASE3
25 participants
6 months
2017-05-15
Participant Flow
Consecutive eligible patients were recruited by the investigators from their clinics and through self-referral between February 2008 and March 2011. 98 persons contacted our study coordinator with queries about the trial, 22 from the Johns Hopkins Interstitial Lung Disease Clinic and 76 in response to the ClinicalTrials.gov Web site.
One participant was excluded because of an FVC\>90% predicted
Participant milestones
| Measure |
Drug Thalidomide First, Then Placebo
Drug Thalidomide 50-100mg daily in the first intervention period and placebo daily in the second intervention period (after washout period)
|
Placebo First, Then Thalidomide Drug
Placebo was administered in the first interventional period and Thalidomide 50-100mg daily in the second interventional period (after washout period).
|
|---|---|---|
|
First Intervention (12 Weeks)
STARTED
|
13
|
11
|
|
First Intervention (12 Weeks)
COMPLETED
|
12
|
10
|
|
First Intervention (12 Weeks)
NOT COMPLETED
|
1
|
1
|
|
Second Intervention (12 Weeks)
STARTED
|
12
|
10
|
|
Second Intervention (12 Weeks)
COMPLETED
|
12
|
8
|
|
Second Intervention (12 Weeks)
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Drug Thalidomide First, Then Placebo
Drug Thalidomide 50-100mg daily in the first intervention period and placebo daily in the second intervention period (after washout period)
|
Placebo First, Then Thalidomide Drug
Placebo was administered in the first interventional period and Thalidomide 50-100mg daily in the second interventional period (after washout period).
|
|---|---|---|
|
First Intervention (12 Weeks)
Withdrawal by Subject
|
1
|
0
|
|
First Intervention (12 Weeks)
Adverse Event
|
0
|
1
|
|
Second Intervention (12 Weeks)
Adverse Event
|
0
|
2
|
Baseline Characteristics
Treatment of Chronic Cough in Idiopathic Pulmonary Fibrosis With Thalidomide
Baseline characteristics by cohort
| Measure |
Drug Thalidomide First, Then Placebo
n=13 Participants
Drug thalidomide 50 - 100 mg daily in the first intervention period and placebo daily in the second intervention period (after washout period)
|
Placebo First, Then Thalidomide Drug
n=11 Participants
Placebo was administered in the first intervention period and Thalidomide 50 - 100 mg daily in the second intervention period(After washout period)
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=39 Participants
|
11 Participants
n=41 Participants
|
24 Participants
n=35 Participants
|
|
Age, Continuous
|
70.5 years
STANDARD_DEVIATION 7.11 • n=39 Participants
|
65.55 years
STANDARD_DEVIATION 7.38 • n=41 Participants
|
67.8 years
STANDARD_DEVIATION 7.8 • n=35 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=39 Participants
|
6 Participants
n=41 Participants
|
14 Participants
n=35 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=39 Participants
|
11 participants
n=41 Participants
|
24 participants
n=35 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: All participants who received the interventions and completed all study visits were included in the efficacy analysis.
The primary endpoint, suppression of cough was measured by the Cough Quality of Life Questionnaire (CQLQ) to measure the effect of interventions on cough-specific quality of life. CQLQ consist of 28 questions about cough and its effects using Likert-like 4-point scales, with lower scores indicating less effect of cough on health related quality of life. CQLQ scale ranges from 28 to 112 ( The lower the value, the higher the quality of life, 28 is considered the best).
Outcome measures
| Measure |
Arm Thalidomide 1st, Placebo 2nd (Intervention Thalidomide)
n=12 Participants
Intervention Thalidomide : thalidomide 50 - 100 mg by mouth daily in the first intervention period
|
Arm Thalidomide 1st, Placebo 2nd (Intervention Placebo)
n=12 Participants
Intervention Placebo : Placebo 50-100 mg by mouth per day in the second intervention period
|
Arm Placebo 1st, Thalidomide 2nd (Intervention Placebo)
n=10 Participants
Intervention Placebo : Placebo 50 - 100 mg by mouth daily in the first intervention period
|
Arm Placebo 1st, Thalidomide 2nd (Intervention Thalidomide)
n=8 Participants
Intervention Thalidomide : thalidomide 50 - 100 mg by mouth daily in the second intervention period
|
|---|---|---|---|---|
|
Efficacy of Thalidomide in Suppressing the Chronic Cough of Idiopathic Pulmonary Fibrosis Using the Cough Quality of Life Questionnaire.
|
47.3 units on a scale
Standard Deviation 13.7
|
55.2 units on a scale
Standard Deviation 14.4
|
61.6 units on a scale
Standard Deviation 11.9
|
44.5 units on a scale
Standard Deviation 12.5
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: All participants who received the interventions and completed all study visits were included in the efficacy analysis.
The secondary endpoint, suppression of cough was measured by the visual analog scale of cough (VAS) was significantly lower during treatment with thalidomide than placebo. Secondary endpoints were Cough VAS - the visual analog scale of cough evaluates the severity of cough in patients with IPF. Visual analog scale of cough ranges from 0 to 100 (0 is considered the best). St. George Respiratory Questionnaire helps to evaluate cough-specific and respiratory quality of life in patients with IPF. St. George Respiratory Questionnaire score ranges from 0 to 100 (0 is considered the best).
Outcome measures
| Measure |
Arm Thalidomide 1st, Placebo 2nd (Intervention Thalidomide)
n=12 Participants
Intervention Thalidomide : thalidomide 50 - 100 mg by mouth daily in the first intervention period
|
Arm Thalidomide 1st, Placebo 2nd (Intervention Placebo)
n=12 Participants
Intervention Placebo : Placebo 50-100 mg by mouth per day in the second intervention period
|
Arm Placebo 1st, Thalidomide 2nd (Intervention Placebo)
n=10 Participants
Intervention Placebo : Placebo 50 - 100 mg by mouth daily in the first intervention period
|
Arm Placebo 1st, Thalidomide 2nd (Intervention Thalidomide)
n=8 Participants
Intervention Thalidomide : thalidomide 50 - 100 mg by mouth daily in the second intervention period
|
|---|---|---|---|---|
|
Efficacy of Thalidomide in Suppressing the Chronic Cough of Idiopathic Pulmonary Fibrosis Using the Visual Analog Scale of Cough and the St. George Respiratory Questionnaire.
Severity of cough (VAS Score)
|
30.3 units on a scale
Standard Deviation 28.5
|
65.9 units on a scale
Standard Deviation 23.3
|
68 units on a scale
Standard Deviation 26.8
|
17.8 units on a scale
Standard Deviation 9.9
|
|
Efficacy of Thalidomide in Suppressing the Chronic Cough of Idiopathic Pulmonary Fibrosis Using the Visual Analog Scale of Cough and the St. George Respiratory Questionnaire.
Quality of life (SGRQ Score)
|
46.4 units on a scale
Standard Deviation 19.4
|
54.7 units on a scale
Standard Deviation 16.9
|
58.8 units on a scale
Standard Deviation 13.3
|
40.7 units on a scale
Standard Deviation 9.5
|
Adverse Events
Thalidomide
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Thalidomide
n=23 participants at risk
Thalidomide : thalidomide 50 - 100 mg by mouth daily
|
Placebo
n=23 participants at risk
Placebo : Placebo 50-100 mg by mouth per day
|
|---|---|---|
|
Gastrointestinal disorders
constipation
|
34.8%
8/23 • Number of events 8
|
4.3%
1/23 • Number of events 1
|
Additional Information
Maureen R. Horton
Johns Hopkins University School of Medicine
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place