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| ID | Type | Description | Link |
|---|---|---|---|
| CG-0747 | Other Grant/Funding Number | Celgene |
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Idiopathic Pulmonary Fibrosis (IPF) is a rapidly progressive lung disorder that is often associated with a chronic, intractable cough. The etiology of the cough associated with IPF is unclear but it is often so severe that it adversely effects the patient's quality of life. We propose that thalidomide specifically suppresses the cough associated with idiopathic pulmonary fibrosis via its anti-inflammatory properties, by suppressing the excessive functional up-regulation of sensory fibers with in the respiratory tract of patients with IPF.
This study is a Phase III, double blinded, randomized, placebo controlled, crossover trial testing the efficacy of thalidomide in suppressing the chronic cough of IPF. The primary objective of this study is to determine the efficacy of thalidomide administered daily for 12 weeks to suppress the chronic cough in patients with idiopathic pulmonary fibrosis as measured by cough specific questionnaires, scales and improved quality of life.
This study is a Phase III, double blinded, randomized, placebo controlled, crossover trial testing the efficacy of thalidomide in suppressing the chronic cough of IPF. All subjects will be randomized to either begin the study receiving the active study drug - (thalidomide) or inactive drug (placebo). Study drug will be administered in escalating dose starting at 50 mg a day increasing to 100 mg a day if cough is still present after 2 weeks. Study drug will be taken by mouth at bedtime. Patients will remain on their initial treatment for 12 weeks. After 12 weeks of treatment, the subjects will be administered the Cough Specific Quality of Life Questionnaire (CQLQ), a visual cough analogue scale, and St. Georges Respiratory Questionnaire (SGRQ). In addition, investigators will collect the subjects cough diary. After 12 weeks of treatment all subjects will enter a 2 week wash-out phase in which all drugs will be discontinued. After the 2 week wash-out phase, all subjects will again be administered the Cough Specific Quality of Life Questionnaire (CQLQ), a visual cough analogue scale, and St. Georges Respiratory Questionnaire (SGRQ). In addition, investigators will collect the subjects cough diary. All subjects will then be crossed over to the other treatment arm for an additional 12 weeks of treatment. After the second 12 weeks of treatment, the subjects will be administered the Cough Specific Quality of Life Questionnaire (CQLQ), a visual cough analogue scale, and St. Georges Respiratory Questionnaire (SGRQ). In addition, investigators will collect the subjects cough diary.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental: Thalidomide, then placebo | Experimental | Participants first received Thalidomide tablet for 12 weeks. After a washout period of two weeks, they then received placebo tablet for 12 weeks. |
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| Experimental: Placebo, then Thalidomide | Experimental | Participants first received Placebo tablet for 12 weeks. After a washout period of two weeks, they then received Thalidomide tablet for 12 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Thalidomide | Drug | Thalidomide 50 - 100 mg by mouth daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of Thalidomide in Suppressing the Chronic Cough of Idiopathic Pulmonary Fibrosis Using the Cough Quality of Life Questionnaire. | 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). | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Maureen Horton, MD | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins Bayview Medical Center | Baltimore | Maryland | 21224 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22986377 | Derived | Horton MR, Santopietro V, Mathew L, Horton KM, Polito AJ, Liu MC, Danoff SK, Lechtzin N. Thalidomide for the treatment of cough in idiopathic pulmonary fibrosis: a randomized trial. Ann Intern Med. 2012 Sep 18;157(6):398-406. doi: 10.7326/0003-4819-157-6-201209180-00003. |
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One participant was excluded because of an FVC>90% predicted
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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) |
| FG001 | 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). |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| First Intervention (12 Weeks) |
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| Second Intervention (12 Weeks) |
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| ID | Title | Description |
|---|---|---|
| BG000 | Drug Thalidomide First, Then Placebo | Drug thalidomide 50 - 100 mg daily in the first intervention period and placebo daily in the second intervention period (after washout period) |
| BG001 | Placebo First, Then Thalidomide Drug |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Efficacy of Thalidomide in Suppressing the Chronic Cough of Idiopathic Pulmonary Fibrosis Using the Cough Quality of Life Questionnaire. | 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). | All participants who received the interventions and completed all study visits were included in the efficacy analysis. | Posted | Mean | Standard Deviation | units on a scale | 6 months |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Thalidomide | Thalidomide : thalidomide 50 - 100 mg by mouth daily |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| constipation | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Maureen R. Horton | Johns Hopkins University School of Medicine | 410-955-4176 | mhorton2@jhmi.edu |
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| ID | Term |
|---|---|
| D054990 | Idiopathic Pulmonary Fibrosis |
| D003371 | Cough |
| ID | Term |
|---|---|
| D011658 | Pulmonary Fibrosis |
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D013792 | Thalidomide |
| ID | Term |
|---|---|
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
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| Placebo | Drug | Placebo 50-100 mg by mouth per day |
|
| 6 months |
| NOT COMPLETED |
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Placebo was administered in the first intervention period and Thalidomide 50 - 100 mg daily in the second intervention period(After washout period)
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG001 | Arm Thalidomide 1st, Placebo 2nd (Intervention Placebo) | Intervention Placebo : Placebo 50-100 mg by mouth per day in the second intervention period |
| OG002 | Arm Placebo 1st, Thalidomide 2nd (Intervention Placebo) | Intervention Placebo : Placebo 50 - 100 mg by mouth daily in the first intervention period |
| OG003 | Arm Placebo 1st, Thalidomide 2nd (Intervention Thalidomide) | Intervention Thalidomide : thalidomide 50 - 100 mg by mouth daily in the second intervention period |
|
|
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| Secondary | 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. | 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). | All participants who received the interventions and completed all study visits were included in the efficacy analysis. | Posted | Mean | Standard Deviation | units on a scale | 6 months |
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|
| 0 |
| 23 |
| 8 |
| 23 |
| EG001 | Placebo | Placebo : Placebo 50-100 mg by mouth per day | 0 | 23 | 1 | 23 |
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| D012120 | Respiration Disorders |
| D012818 | Signs and Symptoms, Respiratory |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D009930 |
| Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| Quality of life (SGRQ Score) |
|